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MX2023008686A - Polinucleotidos moduladores. - Google Patents

Polinucleotidos moduladores.

Info

Publication number
MX2023008686A
MX2023008686A MX2023008686A MX2023008686A MX2023008686A MX 2023008686 A MX2023008686 A MX 2023008686A MX 2023008686 A MX2023008686 A MX 2023008686A MX 2023008686 A MX2023008686 A MX 2023008686A MX 2023008686 A MX2023008686 A MX 2023008686A
Authority
MX
Mexico
Prior art keywords
modulatory polynucleotides
modulatory
polynucleotides
compositions
manufacture
Prior art date
Application number
MX2023008686A
Other languages
English (en)
Inventor
Mathieu E Nonnenmacher
Jinzhao Hou
Pengcheng Zhou
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2023008686A publication Critical patent/MX2023008686A/es

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    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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Abstract

La invención se relaciona con composiciones y métodos para la preparación, la fabricación y el uso terapéutico de polinucleótidos moduladores.
MX2023008686A 2014-11-14 2017-05-12 Polinucleotidos moduladores. MX2023008686A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462079590P 2014-11-14 2014-11-14
US201562212004P 2015-08-31 2015-08-31
US201562234477P 2015-09-29 2015-09-29

Publications (1)

Publication Number Publication Date
MX2023008686A true MX2023008686A (es) 2023-08-01

Family

ID=55955104

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2017006217A MX2017006217A (es) 2014-11-14 2015-11-13 Polinucleotidos moduladores.
MX2023008693A MX2023008693A (es) 2014-11-14 2017-05-12 Polinucleotidos moduladores.
MX2023008686A MX2023008686A (es) 2014-11-14 2017-05-12 Polinucleotidos moduladores.

Family Applications Before (2)

Application Number Title Priority Date Filing Date
MX2017006217A MX2017006217A (es) 2014-11-14 2015-11-13 Polinucleotidos moduladores.
MX2023008693A MX2023008693A (es) 2014-11-14 2017-05-12 Polinucleotidos moduladores.

Country Status (16)

Country Link
US (4) US10570395B2 (es)
EP (2) EP3907287A1 (es)
JP (3) JP6863891B2 (es)
KR (2) KR20230145206A (es)
CN (5) CN112375760A (es)
AU (3) AU2015346164B2 (es)
BR (1) BR112017010088A2 (es)
CA (1) CA2975583C (es)
DK (1) DK3218386T3 (es)
ES (1) ES2878451T3 (es)
IL (2) IL284949B2 (es)
MX (3) MX2017006217A (es)
RU (2) RU2749882C2 (es)
SG (2) SG10202001102XA (es)
WO (1) WO2016077689A1 (es)
ZA (1) ZA202004557B (es)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
KR102652994B1 (ko) * 2016-05-18 2024-04-01 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
SG11201909868YA (en) * 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
TWI897844B (zh) * 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
AU2018261790B2 (en) * 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TW202413649A (zh) * 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
JP2021519065A (ja) * 2018-03-23 2021-08-10 ユニバーシティ オブ マサチューセッツ 骨障害を処置するための遺伝子治療法
CN112601817A (zh) * 2018-05-31 2021-04-02 高丽大学校产学协力团 抑制微小rna的非规范性靶标的诱导rna干扰的核酸及其用途
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US20210361318A1 (en) 2018-07-02 2021-11-25 Voyager Therapeutics, Inc. Cannula system and use thereof
AU2019299861A1 (en) * 2018-07-02 2021-01-14 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
MX2021000810A (es) 2018-07-24 2021-04-28 Voyager Therapeutics Inc Sistemas y metodos para producir formulaciones de terapia genetica.
WO2020023767A1 (en) * 2018-07-26 2020-01-30 Joslin Diabetes Center Targeting micro-rnas for exosomal delivery or cellular retention
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
SG11202103425YA (en) 2018-10-05 2021-05-28 Voyager Therapeutics Inc Engineered nucleic acid constructs encoding aav production proteins
US20210371470A1 (en) * 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
TW202028468A (zh) 2018-10-15 2020-08-01 美商航海家醫療公司 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體
JP2022522995A (ja) 2019-01-18 2022-04-21 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ粒子を生成するための方法及びシステム
AU2020216480A1 (en) 2019-01-31 2021-09-09 Oregon Health & Science University Methods for using transcription-dependent directed evolution of AAV capsids
BR112021020421A2 (pt) * 2019-04-12 2021-12-21 Encoded Therapeutics Inc Composições e métodos para administração de componentes terapêuticos
TW202106879A (zh) 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
AU2020298647A1 (en) * 2019-07-04 2022-02-24 Ractigen Therapeutics Oligomeric nucleic acid molecule activating atoh1 gene and use thereof
US12534725B2 (en) * 2019-08-02 2026-01-27 The Hong Kong University Of Science And Technology Method for controlling microRNA expression
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN111100873B (zh) * 2019-11-22 2020-12-22 昆明学院 激活rna调控启动子克服转基因沉默效应的方法
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202305131A (zh) 2021-02-12 2023-02-01 美商艾拉倫製藥股份有限公司 用於治療或預防超氧歧化酶１－（ＳＯＤ１－）相關的神經退化疾病的超氧歧化酶１（ＳＯＤ１）ｉＲＮＡ組成物及其使用方法
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022225353A1 (ko) * 2021-04-21 2022-10-27 이화여자대학교 산학협력단 타겟 유전자 조절을 위한 다기능성 핵산 구조체 및 이의 용도
KR102897439B1 (ko) * 2021-06-18 2025-12-08 한국과학기술원 타겟 유전자 발현을 조절할 수 있는 핵산 분자 및 이의 용도
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023097268A2 (en) * 2021-11-23 2023-06-01 Mantra Bio, Inc. Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof
CN118946662A (zh) * 2022-03-30 2024-11-12 米瑞莫斯公司 生成新型amiRNA的组合物和方法
US11912997B2 (en) 2022-06-15 2024-02-27 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
AU2023392154A1 (en) * 2022-12-14 2025-06-26 King's College London Compositions and methods for treating neurological diseases
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
CN120202298A (zh) * 2023-08-29 2025-06-24 石药集团中奇制药技术（石家庄）有限公司 一种抑制sod1基因表达的dsRNA分子及其应用
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025126153A2 (en) * 2023-12-14 2025-06-19 Aviadobio Ltd. Compositions and methods for treating sod1-mediated neurological diseases

Family Cites Families (520)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
DE69535703T2 (de) 1994-04-13 2009-02-19 The Rockefeller University AAV-vermittelte Zufuhr von DNA an Zellen des Nervensystems
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
WO1998022588A2 (en) 1996-11-20 1998-05-28 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2304131A1 (en) 1997-09-19 1999-04-01 James M. Wilson Method for gene transfer using bcl2 and compositions useful therein
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
NZ505325A (en) 1997-12-23 2003-07-25 Crucell Holland B Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
AU2882899A (en) 1998-02-26 1999-09-15 Trustees Of The University Of Pennsylvania, The Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
JP2002516345A (ja) 1998-05-27 2002-06-04 セル ジェネシス インコーポレイテッド 第ｖｉｉｉ因子活性のアデノ随伴ウイルスベクター媒介性発現
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
JP4060531B2 (ja) 1998-05-28 2008-03-12 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
US6416992B1 (en) 1998-10-13 2002-07-09 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
CA2348838A1 (en) 1998-10-27 2000-05-04 Crucell Holland B.V. Improved aav vector production
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
EP1183389A2 (en) 1999-04-30 2002-03-06 University of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
JP2003501067A (ja) 1999-06-02 2003-01-14 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法
EP1190249A2 (en) 1999-06-08 2002-03-27 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
WO2001014539A2 (en) 1999-08-20 2001-03-01 Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
US6399385B1 (en) 1999-09-29 2002-06-04 The Trustees Of The University Of Pennsylvania Methods for rapid PEG-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
CA2386546A1 (en) 1999-10-07 2001-04-12 University Of Iowa Research Foundation Adeno-associated viruses and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
WO2001036623A2 (en) 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
IL149667A0 (en) 1999-12-10 2002-11-10 Ariad Gene Therapeutics Inc Un Methods for expression of genes in primates
EP1228234A2 (en) 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
NZ522045A (en) 2000-03-30 2007-05-31 Whitehead Biomedical Inst RNA sequence-specific mediators of RNA interference
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
WO2001083797A2 (en) 2000-04-28 2001-11-08 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
ATE280590T1 (de) 2000-05-23 2004-11-15 Neurologix Inc Glutaminsäure decarboxylase (gad) abgabesystem zur behandlung neurodegeneraliver erkrankungen
WO2001096587A2 (en) 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
US7045299B2 (en) 2000-07-18 2006-05-16 Takeda Pharmaceutical Company Limited Physiologically active peptide and use thereof
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US20020111324A1 (en) 2000-08-17 2002-08-15 Keiya Ozawa Adeno-associated virus-mediated delivery of angiogenic factors
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (es) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
FR2821624B1 (fr) 2001-03-01 2004-01-02 Sod Conseils Rech Applic Nouveau polynucleotide utilisable pour moduler la proliferation des cellules cancereuses
CA2441454A1 (en) 2001-03-14 2002-09-19 Avigen, Inc. Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
ES2375557T3 (es) 2001-06-22 2012-03-02 The Trustees Of The University Of Pennsylvania Adenovirus recombinantes que comprenden prote�?nas de adenovirus de simios y usos de los mismos.
ES2609014T3 (es) 2001-07-12 2017-04-18 University Of Massachusetts Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico
EP1900815B1 (en) 2001-07-12 2016-09-07 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
WO2003014367A1 (en) 2001-08-08 2003-02-20 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
JP4371812B2 (ja) 2001-09-28 2009-11-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ マイクロｒｎａ分子
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
JP4677187B2 (ja) 2001-11-13 2011-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規なアデノ随伴ウイルス（ａａｖ）７配列、それを含むベクターおよびそれらの使用
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
HU230488B1 (hu) 2001-11-21 2016-08-29 The Trustees Of The University Of Pennsylvania Simian adenovírus nukleinsav és aminosav-szekvencia, azt tartalmazó vektorok, és eljárások annak alkalmazására
CA2469623C (en) 2001-12-12 2012-05-29 F H Faulding & Co Limited Composition for the preservation of viruses
WO2003052052A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
EP1463530A4 (en) 2001-12-19 2006-09-06 Lijun Wang ADENO-ASSOCIATED VIRUS-RELATED DELIVERY OF GDNF TO SKELETAL MUSCLES
JP2005538929A (ja) 2002-01-16 2005-12-22 ダイナル バイオテック エイエスエイ 単一サンプルからの核酸及びタンパク質の単離方法
WO2003064665A2 (en) 2002-02-01 2003-08-07 Oxford Biomedica (Uk) Limited Viral vector
US20030180756A1 (en) 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US7235393B2 (en) 2002-04-29 2007-06-26 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues
EP1501921B2 (en) 2002-04-30 2012-07-25 Oncolytics Biotech Inc. Improved viral purification methods
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
DK1504126T3 (da) 2002-05-03 2014-06-10 Univ Duke Fremgangsmåde til regulering af genekspression
PT1506287E (pt) 2002-05-14 2007-07-17 Merck & Co Inc Métodos de purificação de adenovírus
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
EP2801614B1 (en) 2002-08-29 2019-05-15 The Board of Trustees of the Leland Stanford Junior University Circular nucleic acid vectors and methods for making and using the same
AU2003273336A1 (en) 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US20080318210A1 (en) 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
US20060269530A1 (en) 2003-02-21 2006-11-30 The Penn State Research Foundation RNA interference compositions and methods
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
PL2277996T3 (pl) 2003-05-21 2015-03-31 Genzyme Corp Sposoby wytwarzania preparatów rekombinowanych wirionów AAV zasadniczo niezawierających pustych kapsydów
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
HUE034597T2 (en) 2003-06-19 2018-02-28 Genzyme Corp Reduced immunoreactivity of AAV virions and their applications
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
ES2478625T3 (es) 2003-06-20 2014-07-22 The Trustees Of The University Of Pennsylvania Método de generación de adenovirus quiméricos y usos de tales adenovirus quiméricos
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1649028B1 (en) 2003-07-25 2012-08-22 Genvec, Inc. Adenoviral vector-based vaccines
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
EP3760234B1 (en) 2003-09-12 2023-11-01 University of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20050064489A1 (en) 2003-09-24 2005-03-24 Zhang Fang Liang Engineered U6 and H1 promoters
NZ545628A (en) 2003-09-30 2009-04-30 Univ Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
CA2543954C (en) 2003-10-27 2016-11-08 Rosetta Inpharmatics Llc Method of designing sirnas for gene silencing
KR101337579B1 (ko) 2004-03-05 2013-12-06 베니텍 리미티드 ＲＮＡｉ 제제의 동시 전달을 위한 다중 프로모터 발현 카세트
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
EP3290513B1 (en) 2004-06-01 2022-09-07 Genzyme Corporation Compositions and methods to prevent aav vector aggregation
DK1807009T3 (en) 2004-10-05 2015-03-02 Genzyme Corp Stair shaped needle
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
PT2189469E (pt) 2004-11-18 2016-01-22 Univ Illinois Construções de arnsi multicistrónicas para inibir tumores
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
WO2006066203A2 (en) 2004-12-16 2006-06-22 Alsgen, Llc Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod)
NZ560936A (en) 2005-02-03 2010-04-30 Benitec Inc RNAI expression constructs
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
US8999943B2 (en) 2005-03-14 2015-04-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
EP2359866B1 (en) 2005-04-07 2013-07-17 The Trustees of The University of Pennsylvania Modified AAV rh48 capsids, compositions containing same and uses thereof
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
ES2887076T3 (es) 2005-05-02 2021-12-21 Genzyme Corp Terapia génica para trastornos neurometabólicos
US20070105803A1 (en) 2005-08-18 2007-05-10 Muthiah Manoharan Methods and compositions for treating neurological disease
AU2006283189B2 (en) 2005-08-23 2013-01-31 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
CN101287837B (zh) 2005-10-20 2015-03-18 尤尼克尔生物制药股份有限公司 昆虫细胞中生产的改进的aav载体
RU2448974C2 (ru) 2005-11-01 2012-04-27 Элнилэм Фармасьютикалз, Инк. РНКи-ИНГИБИРОВАНИЕ РЕПЛИКАЦИИ ВИРУСА ГРИППА
WO2007086990A2 (en) 2005-11-17 2007-08-02 Board Of Regents, The University Of Texas System Modulation of gene expression by oligomers targeted to chromosomal dna
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CA3024953A1 (en) * 2006-04-03 2007-10-11 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
WO2007127264A2 (en) 2006-04-28 2007-11-08 The Trustees Of The University Of Pennsylvania Scalable production method for aav
JP5889514B2 (ja) 2006-04-28 2016-03-22 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 改変アデノウイルスヘキソンタンパク質およびその用途
WO2007130519A2 (en) 2006-05-02 2007-11-15 Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
CA2651499C (en) 2006-05-25 2015-06-30 Sangamo Biosciences, Inc. Methods and compositions for ccr-5 gene inactivation
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
EP2048955B1 (en) 2006-07-21 2013-08-21 California Institute of Technology Targeted gene delivery for dendritic cell vaccination
EP2061891B1 (en) 2006-08-24 2012-04-11 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
SI2497500T1 (sl) 2006-10-03 2017-01-31 Genzyme Corporation Genska terapija za spinalno mišično atrofijo
EP2679237A1 (en) 2006-11-29 2014-01-01 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
EP2647711B1 (en) 2006-11-29 2018-01-10 University of Iowa Research Foundation Alternative export pathways for vector expressed RNA interference
US8071752B2 (en) 2007-01-29 2011-12-06 City Of Hope Multi-targeting short interfering RNAs
MX2009008178A (es) 2007-02-02 2009-10-26 Biogen Idec Inc Uso de semaforina 6a para promover la mielinizacion y la diferenciacion de oligodendrocitos.
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
CA2720097C (en) 2007-04-09 2016-07-19 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
CA2688514A1 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2167521A4 (en) * 2007-06-15 2011-11-23 Univ Ohio State Res Found ONKOGEN ALL-1 FUSION PROTEINS FOR TARGETING DROSHA-MEDIATED microRNA PROCESSING
US8841437B2 (en) 2008-06-20 2014-09-23 The Board Of Trustees Of The Leland Stanford Junior University Precursor miRNA loop-modulated target regulation
CA2728571A1 (en) * 2007-06-29 2009-01-08 Boston Biomedical, Inc. Bacteria-mediated gene modulation via microrna machinery
KR101183764B1 (ko) 2007-06-29 2012-09-17 에프. 호프만-라 로슈 아게 프로모터
CA2693712C (en) 2007-07-14 2018-05-29 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
DK3093345T3 (da) 2007-07-26 2019-06-24 Uniqure Ip Bv Baculovirusvektorer omfattende gentagne kodende sekvenser med differentielle foretrukne kodoner
JP2010537639A (ja) 2007-08-27 2010-12-09 ボストン バイオメディカル， インコーポレイテッド 非対称性干渉ｒｎａの組成物およびその使用
CA2698011C (en) 2007-09-04 2015-12-29 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Health Porcine adeno-associated viruses
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
EP2205740A2 (en) 2007-10-02 2010-07-14 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
EP2205746A4 (en) 2007-10-04 2010-12-22 Univ Texas MODULATION OF GENE EXPRESSION WITH AGRNA AND GAPS WITH ANTISENSE TRANSCRIPTS AS A TARGET
EP2186283A4 (en) 2007-10-18 2011-03-09 Lg Electronics Inc METHOD AND SYSTEM FOR TRANSMITTING AND RECEIVING SIGNALS
ES2607029T3 (es) 2007-11-28 2017-03-28 The Trustees Of The University Of Pennsylvania Adenovirus que comprende una proteína hexónica de la cápside del adenovirus E de simio SAdV-39 y usos de la misma
ES2621165T3 (es) 2007-11-28 2017-07-03 The Trustees Of The University Of Pennsylvania Adenovirus de simio de la subfamilia B SADV-28, -27, -29, -32, -33 y -35 y usos de los mismos
BRPI0819774A2 (pt) 2007-11-28 2014-10-14 Univ Pennsylvania Subfamília c de adenovírus sadv-40, -31 e -34 de símio e seus usos
WO2009086428A2 (en) 2007-12-28 2009-07-09 The Regents Of The University Of California Methods and compositions for increasing gene expression
US8133488B2 (en) 2008-01-18 2012-03-13 Genentech, Inc. Methods and compositions for targeting polyubiquitin
AU2009209408B2 (en) 2008-01-29 2015-06-11 Beacon Therapeutics Limited Recombinant virus production using mammalian cells in suspension
AU2009213147A1 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified RNAi polynucleotides and uses thereof
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
JP5661476B2 (ja) 2008-03-04 2015-01-28 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア サルアデノウイルスＳＡｄＶ−３６、−４２．１、−４２．２および−４４ならびにそれらの用途
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US20120093772A1 (en) 2008-05-20 2012-04-19 Alan Horsager Vectors for delivery of light sensitive proteins and methods of use
JP5689413B2 (ja) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 平滑末端を有する５’三リン酸オリゴヌクレオチドおよびその使用
US9095126B2 (en) 2008-05-27 2015-08-04 Yale University Targeting TGF-β as a therapy for Alzheimer's disease
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
WO2009151620A2 (en) 2008-06-13 2009-12-17 Cornell Research Foundation, Inc. Wmc Pain treatment using erk2 inhibitors
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
WO2010002851A1 (en) 2008-06-30 2010-01-07 Alnylam Pharmaceuticals, Inc. Silencing and rig-1 activation by dual function oligonucleotides
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
CA2731779A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US8212019B2 (en) 2008-07-30 2012-07-03 University Of Massachusetts Nucleic acid silencing sequences
CN102405286A (zh) 2008-09-22 2012-04-04 阿克赛医药公司 减小大小的自递送RNAi化合物
KR20110086553A (ko) 2008-09-29 2011-07-28 암스테르담 몰레큘러 테라퓨틱스 비. 브이. 포르포빌리노겐 디아미나아제 유전자 치료
KR20110081862A (ko) 2008-10-22 2011-07-14 제넨테크, 인크. 축삭 변성의 조절
WO2010051367A1 (en) 2008-10-31 2010-05-06 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-43, -45,-48,-49, and -50 and uses thereof
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
MX2011009193A (es) 2009-03-04 2011-12-16 Deutsches Krebsforsch Proteina activadora del ensamblaje (aap) y su uso para la fabricacion de particulas de parvovirus que consisten esencialmente de vp3.
CN102448501A (zh) 2009-03-27 2012-05-09 西马生物医学计划公司 治疗肝硬化和肝纤维化的方法和组合物
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
WO2010136549A2 (en) 2009-05-28 2010-12-02 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CA2762203A1 (en) 2009-05-29 2010-12-02 Soumitra Roy Simian adenovirus 41 and uses thereof
US20120142764A1 (en) 2009-06-05 2012-06-07 Dai-Wu Seol Multi-Cistronic shRNA Expression Cassette for Suppressing Single or Multiple Target Genes
DK3067417T3 (en) 2009-06-16 2018-11-19 Genzyme Corp IMPROVED PROCEDURES FOR CLEANING RECOMBINANT AAV VECTORS
CA2767231A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
US20140099666A1 (en) 2009-07-06 2014-04-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
WO2011008348A2 (en) 2009-07-15 2011-01-20 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
WO2011054976A2 (en) 2009-11-09 2011-05-12 Genepod Therapeutics Ab Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo
EP2508603B1 (en) 2009-11-19 2015-05-13 National University Corporation Okayama University System for increasing gene expression, and vector supporting said system
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2011101869A1 (en) * 2010-02-22 2011-08-25 Transgene Biotek Ltd. Adeno-associated virus 2/8 - micro rna-101 therapy for liver cancer
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
EP3536781A1 (en) 2010-04-23 2019-09-11 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2601293B1 (en) 2010-08-02 2017-12-06 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2012018881A2 (en) 2010-08-03 2012-02-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for the regulation of rna
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN101972476B (zh) * 2010-09-14 2012-12-19 中国人民解放军第二军医大学 利用MicroRNA-155的核酸疫苗佐剂及其构建方法
JP5010760B2 (ja) 2010-10-05 2012-08-29 タカラバイオ株式会社 ウイルスベクターの製造方法
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CN103189507A (zh) 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
WO2012064920A1 (en) 2010-11-11 2012-05-18 University Of Miami Methods, compositions, cells, and kits for treating ischemic injury
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
EP2673286B1 (en) 2011-02-12 2019-07-03 University of Iowa Research Foundation Therapeutic compounds
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US9610354B2 (en) 2011-04-18 2017-04-04 National Center Of Neurology And Psychiatry Drug delivery particle and method for producing the same
EP2699688A1 (en) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
CA2870511C (en) 2011-04-21 2023-08-08 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
SI3693025T1 (sl) 2011-04-22 2022-04-29 The Regents Of The University Of California Virioni adeno-povezanega virusa z varianto kapsida in postopki za njihovo uporabo
WO2012149646A1 (en) * 2011-05-05 2012-11-08 Sunnybrook Research Institute Mirna inhibitors and their uses
WO2012158757A1 (en) 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
US9598468B2 (en) 2011-05-18 2017-03-21 University Of Florida Research Foundation, Incorporated Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof
EP2718713B1 (en) 2011-06-06 2019-07-17 Koninklijke Philips N.V. Selective lysis of cells by ionic surfactants
EP4488370A2 (en) 2011-07-25 2025-01-08 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expresssion of dux4
DK2737071T3 (en) 2011-07-27 2017-05-08 Genethon IMPROVED BACULOVIRUS EXPRESSION SYSTEMS
US8852911B2 (en) 2011-08-04 2014-10-07 The Regents Of The University Of California Method of producing dicer
WO2013033627A2 (en) 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
HUE026579T2 (en) 2011-09-08 2016-06-28 Uniqure Ip Bv Removal of virus infections from AAV preparations
US9056892B2 (en) 2011-09-09 2015-06-16 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
US9464322B2 (en) 2011-09-09 2016-10-11 University Of Kentucky Research Foundation Methods for diagnosing and treating alzheimer's disease (AD) using the molecules that stabilize intracellular calcium (Ca2+) release
SG11201400125RA (en) 2011-09-19 2014-03-28 Axon Neuroscience Se Protein-based therapy and diagnosis of tau-mediated pathology in alzheimer's disease
CN104520421B (zh) 2011-10-28 2017-05-24 北卡罗来纳-查佩尔山大学 用于生产腺伴随病毒的细胞系
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
WO2013103896A1 (en) 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
WO2013113696A1 (en) 2012-01-30 2013-08-08 Vib Vzw Means and method for diagnosis and treatment of alzheimer's disease
DK2814513T3 (en) 2012-02-14 2018-02-26 Univ California Systemic administration and regulated expression of paracrine for cardiovascular disease and other conditions
WO2013122605A1 (en) 2012-02-17 2013-08-22 Evernote Corporation Site memory processing
US20150111955A1 (en) 2012-02-17 2015-04-23 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
US20150148405A1 (en) 2012-02-21 2015-05-28 The Johns Hopkins University Expression Construct for a Lin28-Resistant Let-7 Precursor MicroRNA
RU2639277C2 (ru) 2012-02-29 2017-12-20 Сангамо Байосайенсиз, Инк. Способы и составы лечения болезни хантингтона
HUE054087T2 (hu) 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
CA2871920C (en) 2012-05-08 2021-03-09 Merck Sharp & Dohme Corp. Permeable glycosidase inhibitors and uses thereof
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2850194A4 (en) 2012-05-18 2016-06-08 Univ Pennsylvania APE-ADENOVIRES A1302, A1320, A1331 AND A1337 OF THE FAMILY E AND USES THEREOF
BR112014028666B1 (pt) 2012-05-18 2021-12-07 The General Hospital Corporation Uso de uma partícula de raav2, composição farmacêutica e kit
US9539259B2 (en) 2012-05-23 2017-01-10 The Johns Hopkins University Compounds and methods of use thereof for treating neurodegenerative disorders
BR112015000161A2 (pt) 2012-07-06 2017-06-27 Univ Iowa Res Found composições de vetor de vírus adeno-associado modificado
WO2014007120A1 (ja) 2012-07-06 2014-01-09 タカラバイオ株式会社 アデノ随伴ウイルスベクターの産生細胞
EP2875133B1 (en) * 2012-07-17 2018-01-10 Université de Genève Nucleic acids for down-regulation of gene expression
JP6314138B2 (ja) 2012-08-01 2018-04-18 ネイションワイド チルドレンズ ホスピタル 組換えアデノ随伴ウイルス９の髄腔内送達
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
EP3738974A1 (en) 2012-09-28 2020-11-18 The University of North Carolina at Chapel Hill Aav vectors targeted to oligodendrocytes
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
WO2014071042A1 (en) 2012-10-31 2014-05-08 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
CN104937100B (zh) 2012-12-25 2020-04-03 宝生物工程株式会社 Aav 变体
SG11201505272QA (en) * 2013-01-08 2015-08-28 Benitec Biopharma Ltd Age-related macular degeneration treatment
CA2897444A1 (en) 2013-01-08 2014-07-17 Genzyme Corporation Use of inos inhibitors to increase viral yield in culture
US9066966B2 (en) * 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
US9562429B2 (en) 2013-03-12 2017-02-07 Halliburton Energy Services, Inc. Wellbore servicing tools, systems and methods utilizing near-field communication
WO2014160092A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
WO2014144844A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University tRNA DERIVED SMALL RNAs (tsRNAs) INVOLVED IN CELL VIABILITY
BR112015021884A8 (pt) 2013-03-15 2019-11-26 Childrens Hospital Philadelphia vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica
CA2909085C (en) 2013-04-08 2023-08-29 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
JP6461917B2 (ja) 2013-04-20 2019-01-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル エクソン２標的Ｕ７ｓｎＲＮＡポリヌクレオチド構築物の組換えアデノ随伴ウイルスによる送達
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
BR112015028605A8 (pt) 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2014201308A1 (en) 2013-06-12 2014-12-18 Washington University Endothelial-targeted adenoviral vectors, methods and uses therefor
WO2014201252A2 (en) 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
JP6752142B2 (ja) 2013-07-26 2020-09-09 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 脳疾患を処置するための方法および組成物
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
CA2922500A1 (en) 2013-08-27 2015-03-05 Research Institute At Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
LT3459965T (lt) 2013-10-11 2021-03-10 Massachusetts Eye & Ear Infirmary Viruso prototipo sekų ir jų panaudojimo prognozavimo metodai
EP3060669B1 (en) 2013-10-24 2024-12-11 uniQure IP B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
JP2016538276A (ja) 2013-11-05 2016-12-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔ Ｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のためのＮＦ−κＢおよびＳＯＤ−１を阻害する組成物および方法
EP3066195A4 (en) 2013-11-08 2017-06-28 The Board of Trustees of the University of Arkansas Adeno-associated virus "x" oncogene
KR102431079B1 (ko) 2013-11-11 2022-08-11 상가모 테라퓨틱스, 인코포레이티드 헌팅턴병을 치료하기 위한 방법 및 조성물
ES2690643T3 (es) 2013-11-26 2018-11-21 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Vectores virales adeno-asociados para el tratamiento de enfermedad de almacenamiento de glucógeno
US10161011B2 (en) 2013-11-29 2018-12-25 Takara Bio Inc. Method for quantifying adeno-associated virus
US20170002350A1 (en) 2013-12-03 2017-01-05 Agency For Science, Technology And Research Tailed mirtron effectors for rnai-mediated gene silencing
JP6707028B2 (ja) 2013-12-09 2020-06-10 ベイラー カレッジ オブ メディスンＢａｙｌｏｒ Ｃｏｌｌｅｇｅ Ｏｆ Ｍｅｄｉｃｉｎｅ 心筋細胞新生におけるｈｉｐｐｏ及びジストロフィン複合体シグナル伝達
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
US20150197809A1 (en) 2014-01-13 2015-07-16 Trustees Of Boston University Methods and assays relating to huntingtons disease and parkinson's disease
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
WO2015124540A1 (en) 2014-02-19 2015-08-27 F. Hoffmann-La Roche Ag Blood brain barrier shuttle
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
WO2015127351A1 (en) 2014-02-24 2015-08-27 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015134920A1 (en) 2014-03-07 2015-09-11 The Arizona Board Of Regents On Behalf Of The University Of Arizona Non-narcotic crmp2 peptides targeting sodium channels for chronic pain
AU2015230094B2 (en) 2014-03-10 2021-05-27 Uniqure Ip B.V. Further improved AAV vectors produced in insect cells
EP3119797B1 (en) 2014-03-18 2020-12-23 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
SG10201910844SA (en) 2014-04-01 2020-01-30 Biogen Ma Inc Compositions for modulating sod-1 expression
EP3556858A3 (en) 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US20170088819A1 (en) 2014-05-16 2017-03-30 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
US9523093B2 (en) 2014-05-20 2016-12-20 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
JP7026440B2 (ja) 2014-05-28 2022-02-28 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ハイブリッドｔＲＮＡ／プレｍｉＲＮＡ分子および使用方法
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
WO2015191780A2 (en) 2014-06-10 2015-12-17 The General Hospital Corporation Ccctc-binding factor (ctcf) rna interactome
US11207424B2 (en) 2014-06-13 2021-12-28 Mayo Foundation For Medical Education And Research Methods and materials for increasing viral vector infectivity
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
US10526583B2 (en) 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
US10023846B2 (en) 2014-07-10 2018-07-17 Takara Bio Inc. Production method for non-enveloped virus particles
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
CA2955285C (en) 2014-07-31 2023-09-26 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
EP3191593A4 (en) 2014-09-08 2018-01-24 University of Iowa Research Foundation Microrna inhibitor system and methods of use thereof
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
EP3215602B1 (en) 2014-11-05 2019-12-25 The Research Institute at Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
KR20230145206A (ko) * 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
CA2967393A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
EP3591070B1 (en) 2014-11-28 2022-12-21 uniQure IP B.V. Dna impurities in a composition comprising a parvoviral virion
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EA037696B1 (ru) 2014-12-24 2021-05-12 ЮНИКЕР АйПи Б.В. Супрессия гена гентингтина, индуцированная рнк-интерференцией
WO2016109649A1 (en) 2014-12-30 2016-07-07 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CA3221229A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180008727A1 (en) 2015-01-30 2018-01-11 The Regents Of The University Of California Spinal subpial gene delivery system
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
NZ772772A (en) 2015-02-10 2024-12-20 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US20180094280A1 (en) 2015-03-20 2018-04-05 Bluebird Bio, Inc. Vector formulations
AU2016235163B2 (en) 2015-03-24 2022-03-24 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2016161374A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CN108093639B (zh) 2015-04-16 2022-07-19 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US20160346359A1 (en) 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
CA2985223A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
CN107735140B (zh) 2015-05-11 2021-04-20 亚克安娜生命科学有限公司 药物输送系统和方法
EP3294894B8 (en) 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10729790B2 (en) 2015-05-26 2020-08-04 Salk Institute For Biological Studies Motor neuron-specific expression vectors
CN108271364A (zh) 2015-05-29 2018-07-10 宾夕法尼亚大学理事会 用于降解错误折叠的蛋白质的组合物和方法
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
WO2017004514A1 (en) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
ES2895652T3 (es) 2015-07-07 2022-02-22 Inst Nat Sante Rech Med Métodos y composiciones farmacéuticas para expresar un polinucleótido de interés en el sistema nervioso periférico de un sujeto
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
JP7166168B2 (ja) 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
WO2017024111A1 (en) 2015-08-04 2017-02-09 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
US10047377B2 (en) 2015-09-22 2018-08-14 Loyola University Of Chicago Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
JP6853257B2 (ja) 2015-09-23 2021-03-31 サンガモ セラピューティクス， インコーポレイテッド Ｈｔｔリプレッサー及びその使用
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
AU2016335699A1 (en) 2015-10-09 2018-05-24 Genzyme Corporation Early post-transfection isolation of cells (EPIC) for biologics production
EP3359662A4 (en) 2015-10-09 2019-06-19 The Children's Hospital of Philadelphia COMPOSITIONS AND METHODS OF TREATING HUNTINGTON DISEASE AND RELATED DISEASES
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3364970B1 (en) 2015-10-23 2024-02-07 University of Iowa Research Foundation Gene therapy for use in treating lysosomal storage disease
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
BR112018008407A2 (en) 2015-10-28 2018-11-27 The Trustees Of The University Of Pennsylvania intrathecal administration of adeno-associated viral vectors for gene therapy
WO2017079768A1 (en) 2015-11-08 2017-05-11 Genentech, Inc. Methods of screening for multispecific antibodies
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
US20170151416A1 (en) 2015-12-01 2017-06-01 Invivo Therapeutics Corporation Methods and Systems for Delivery of a Trail of a Therapeutic Substance into an Anatomical Space
CO2018006699A2 (es) 2015-12-01 2018-09-20 Spark Therapeutics Inc Métodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspensión sin suero adecuado para uso clínico
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US11371061B2 (en) 2015-12-03 2022-06-28 Genethon Compositions and methods for improving viral vector efficiency
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
EP3387137B1 (en) 2015-12-11 2021-02-03 California Institute of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
EP4215605A1 (en) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Scalable purification method for aav8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
JP6924441B2 (ja) 2016-01-15 2021-08-25 学校法人自治医科大学 てんかん治療のためのアデノ随伴ウイルスビリオン
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
JP2019504888A (ja) 2016-02-05 2019-02-21 エモリー ユニバーシティ 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
AU2017219865B2 (en) 2016-02-16 2023-04-13 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
US20190071681A1 (en) 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
MX2018010842A (es) 2016-03-07 2019-07-04 Univ Iowa Res Found Expresion mediada por el virus adeno-asociado (aav) usando un promotor y pontenciador sintetico.
EP3429605A4 (en) 2016-03-18 2019-09-18 The Children's Hospital of Philadelphia THERAPEUTIC FOR THE TREATMENT OF DISEASES INCLUDING THE CENTRAL NERVOUS SYSTEM
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
DK3436593T3 (da) 2016-03-28 2023-02-20 Ultragenyx Pharmaceutical Inc Fremgangsmåder til varmeinaktivering af adenovirus
WO2017172733A1 (en) 2016-03-28 2017-10-05 The Regents Of The University Of California Anti-ryk antibodies and methods of using the same
EP3436576A4 (en) 2016-03-30 2019-10-30 Spark Therapeutics, Inc. CELL LINE FOR RECOMBINANT PROTEIN AND / OR VIRUS VECTOR PRODUCTION
PT3436051T (pt) 2016-03-31 2021-11-02 Spark Therapeutics Inc Processo de fabrico de raav com base em coluna totalmente escalável
JP7140683B2 (ja) 2016-04-15 2022-09-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規なａａｖ８変異カプシド及びそれを含有する組成物
BR112018071180A2 (pt) 2016-04-16 2019-02-12 University Of Florida Research Foundation, Incorporated métodos de reforço potência biológica de baculovirus system-produzido vírus adeno-associado recombinante
IL261665B2 (en) 2016-04-21 2023-11-01 Virovek Inc AVV production in insect cells, methods and preparations thereof
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
US10585103B2 (en) 2016-04-28 2020-03-10 The Trustees Of Indiana University Methods and compositions for resolving components of a virus preparation
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US10882886B2 (en) 2016-05-03 2021-01-05 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
KR102652994B1 (ko) * 2016-05-18 2024-04-01 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
WO2018191450A2 (en) 2017-04-14 2018-10-18 National Taiwan University Hospital Gene therapy for aadc deficiency
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
TWI897844B (zh) 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
AU2018261790B2 (en) * 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN118581154A (zh) 2017-06-02 2024-09-03 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
GB201714027D0 (en) 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2019299861A1 (en) 2018-07-02 2021-01-14 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord

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