[go: up one dir, main page]

LUC00046I2 - - Google Patents

Download PDF

Info

Publication number
LUC00046I2
LUC00046I2 LU00046C LUC00046C LUC00046I2 LU C00046 I2 LUC00046 I2 LU C00046I2 LU 00046 C LU00046 C LU 00046C LU C00046 C LUC00046 C LU C00046C LU C00046 I2 LUC00046 I2 LU C00046I2
Authority
LU
Luxembourg
Application number
LU00046C
Original Assignee
Cold Spring Harbor Laboratory
Biogen Ma 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 Cold Spring Harbor Laboratory, Biogen Ma Inc filed Critical Cold Spring Harbor Laboratory
Publication of LUC00046I1 publication Critical patent/LUC00046I1/fr
Publication of LUC00046I2 publication Critical patent/LUC00046I2/fr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
LU00046C 2005-06-23 2017-11-07 LUC00046I2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (fr) 2005-06-23 2006-06-23 Compositions et procédés de modulation de l épissage de SMN2

Publications (2)

Publication Number Publication Date
LUC00046I1 LUC00046I1 (fr) 2017-11-10
LUC00046I2 true LUC00046I2 (fr) 2018-01-10

Family

ID=37595872

Family Applications (1)

Application Number Title Priority Date Filing Date
LU00046C LUC00046I2 (fr) 2005-06-23 2017-11-07

Country Status (13)

Country Link
US (6) US8361977B2 (fr)
EP (5) EP1910395B1 (fr)
CY (2) CY2017040I2 (fr)
DK (3) DK3308788T3 (fr)
ES (3) ES2545223T3 (fr)
HK (1) HK1247103B (fr)
HU (2) HUE027486T2 (fr)
LT (2) LT3308788T (fr)
LU (1) LUC00046I2 (fr)
PL (3) PL3308788T3 (fr)
PT (3) PT2548560E (fr)
SI (2) SI3308788T1 (fr)
WO (1) WO2007002390A2 (fr)

Families Citing this family (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
CA2596506C (fr) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Composition antisens et methode permettant de traiter une atrophie musculaire
PL3308788T3 (pl) 2005-06-23 2019-05-31 Biogen Ma Inc Kompozycje i sposoby modulacji splicingu smn2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
US8637478B2 (en) 2007-11-13 2014-01-28 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009151546A2 (fr) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Procédés de traitement d'une atrophie musculaire spinale
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
IT1398361B1 (it) * 2009-01-21 2013-02-22 Fond Santa Lucia Uso di mutanti dominanti negativi di sam68 per il trattamento della sma
DK2417257T3 (da) * 2009-04-10 2016-06-06 Ass Inst De Myologie Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom
WO2010120820A1 (fr) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions et procédés pour moduler l'épissage de smn2
PT3449926T (pt) * 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
KR101885383B1 (ko) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 신규한 핵산 프로드러그 및 그의 사용 방법
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
WO2011150408A2 (fr) 2010-05-28 2011-12-01 Avi Biopharma, Inc. Analogues oligonucléotidiques ayant des liaisons modifiées entre sous-unités et/ou des groupes terminaux modifiés
WO2011159836A2 (fr) * 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Composés et procédés pour moduler l'interaction entre des protéines et des acides nucléiques cibles
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
IT1405793B1 (it) * 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
ES2633565T3 (es) 2010-11-12 2017-09-22 The General Hospital Corporation ARN no codificantes asociados a polycomb
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
IL273838B (en) 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
WO2012178122A2 (fr) * 2011-06-23 2012-12-27 Cold Spring Harbor Laboratory Modèle phénocopique de maladie
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EA201400566A1 (ru) 2011-11-11 2014-09-30 Сэнтерис Фарма А/С Соединения, предназначенные для модуляции сплайсинга smn2
EA202090338A1 (ru) 2011-11-18 2021-04-30 Сарепта Терапьютикс, Инк. Функционально-модифицированные олигонуклеотиды и их субъединицы
EP3858847A1 (fr) 2011-11-30 2021-08-04 Sarepta Therapeutics, Inc. Oligonucléotides pour le traitement de maladies à répétition nucléotidique étendue
WO2013082551A1 (fr) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Inclusion d'exon induite pour l'amyotrophie spinale
EP2788087A4 (fr) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Agents de contraste non ioniques de faible osmolarité pour l'administration d'oligonucléotides antisens et le traitement de maladie
CN112251436A (zh) 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
KR20150030205A (ko) 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
JP2015518713A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Ｕｔｒｎ発現を調節するための組成物及び方法
US20150152410A1 (en) 2012-05-16 2015-06-04 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
RU2693381C2 (ru) 2012-07-13 2019-07-02 Уэйв Лайф Сайенсес Лтд. Асимметричная вспомогательная группа
WO2014012081A2 (fr) 2012-07-13 2014-01-16 Ontorii, Inc. Contrôle chiral
WO2014110291A1 (fr) * 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet
US9856474B2 (en) 2013-01-16 2018-01-02 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
CN105793422B (zh) 2013-09-05 2020-03-03 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
EP3052632A4 (fr) * 2013-10-04 2017-03-29 Rana Therapeutics, Inc. Compositions et méthodes pour traiter la sclérose latérale amyotrophique (sla)
WO2015108048A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
EP3095461A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
WO2015120450A1 (fr) * 2014-02-10 2015-08-13 Ohio State Innovation Foundation Oligonucléotides antisens dans le traitement de l'amyotrophie spinale
WO2015161170A2 (fr) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions et procédés de modulation de l'épissage du smn2 chez un patient
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016040748A1 (fr) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions et procédés de détection d'une protéine smn chez un patient et traitement d'un patient
WO2016054615A2 (fr) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Augmentation ciblée de la production de gènes nucléaires
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016164896A2 (fr) 2015-04-10 2016-10-13 Ionis Pharmaceuticals, Inc. Modulation de l'expression de smn
EP3851531A1 (fr) 2015-06-01 2021-07-21 Sarepta Therapeutics, Inc. Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
WO2016196897A1 (fr) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Procédés et composés pour le traitement de maladies et d'états pathologiques associés aux lymphocytes
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
EA201890908A1 (ru) 2015-10-09 2018-10-31 Сарепта Терапьютикс, Инк. Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушений
IL308174A (en) 2015-10-09 2024-01-01 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
US9669109B1 (en) 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
WO2017136435A1 (fr) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Composés permettant de moduler l'expression de fc-epsilon-ri-bêta et utilisations correspondantes
EP3445405A4 (fr) 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. Oligomères antisens et procédés d'utilisation de ceux-ci pour le traitement de maladies associées au gène de l'alpha-glucosidase acide
ES2988362T3 (es) 2016-06-14 2024-11-20 Biogen Ma Inc Cromatografía de interacción hidrófoba para la purificación de oligonucleótidos
WO2017218884A1 (fr) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinaisons pour la modulation de l'expression de smn
JP7235512B2 (ja) 2016-06-24 2023-03-08 バイオジェン・エムエイ・インコーポレイテッド キャッピング工程を有さないチオール化オリゴヌクレオチドの合成
EP3481432A4 (fr) 2016-07-05 2020-05-06 The U.S.A. as represented by the Secretary, Department of Health and Human Services Diagnostic de maladies associées au gène col6 et procédés de traitement associés
AU2017297624B2 (en) * 2016-07-15 2023-11-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
EP3548005A4 (fr) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes destinés à l'administration d'agents thérapeutiques
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
AU2018256435B2 (en) 2017-04-20 2025-03-13 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
IL321831A (en) 2017-08-25 2025-08-01 Stoke Therapeutics Inc Antisense oligomers for the treatment of conditions and diseases
JP2020537518A (ja) 2017-10-12 2020-12-24 ウェーブ ライフ サイエンシーズ リミテッド オリゴヌクレオチド組成物及びその方法
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
IL314692A (en) 2017-11-08 2024-10-01 Novartis Ag Means and method for preparing viral vectors and their uses
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2019147960A1 (fr) 2018-01-25 2019-08-01 Biogen Ma Inc. Méthodes de traitement de l'amyotrophie musculaire
BR112020022512A2 (pt) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. métodos e composições para tratamento de doença de armazenamento de éster de colesteril
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
KR20210111242A (ko) 2018-10-29 2021-09-10 바이오젠 엠에이 인코포레이티드 혈액 뇌 장벽 수송을 향상시키기 위한 인간화 및 안정화된 fc5 변이체
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
TWI844587B (zh) 2018-11-30 2024-06-11 瑞士商諾華公司 Aav病毒載體及其用途
MA54383A (fr) 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
US20220154182A1 (en) 2019-03-20 2022-05-19 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
IL319933A (en) 2019-05-08 2025-05-01 Biogen Ma Inc Convergent Syntheses of Oligonucleotides in Liquid Phase
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal
CN115279379B (zh) 2020-02-28 2025-05-09 Ionis制药公司 用于调节smn2的化合物和方法
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
JP2024518068A (ja) 2021-05-10 2024-04-24 エントラーダ セラピューティクス，インコーポレイティド 細胞内治療のための組成物及び方法
CN118043039A (zh) 2021-10-25 2024-05-14 诺华股份有限公司 用于改善腺相关病毒（aav）递送的方法
FR3128873B1 (fr) 2021-11-10 2026-01-16 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale
WO2023240236A1 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale
WO2025035143A1 (fr) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement de l'amyotrophie spinale

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
FR2645345A1 (fr) * 1989-03-31 1990-10-05 Thomson Csf Procede de modulation dirigee de la composition ou du dopage de semi-conducteurs, notamment pour la realisation de composants electroniques monolithiques de type planar, utilisation et produits correspondants
US5256775A (en) * 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
DK0804219T3 (da) 1994-02-28 2002-11-11 Agritech Technologies Ltd Inhibinpræparater og fremgangsmåder til anvendelse deraf
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20030228597A1 (en) * 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6172216B1 (en) * 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6214986B1 (en) * 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
DE19906484A1 (de) * 1999-02-17 2000-09-07 Wegerer Kristina Elektrische Maschine
DK1178999T3 (da) * 1999-05-04 2007-08-06 Santaris Pharma As L-RIBO-LNA-analoger
US6649411B2 (en) 1999-07-30 2003-11-18 The United States Of America As Represented By The Department Of Health And Human Services Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides
US6770633B1 (en) 1999-10-26 2004-08-03 Immusol, Inc. Ribozyme therapy for the treatment of proliferative skin and eye diseases
US6962906B2 (en) * 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6376508B1 (en) * 2000-12-13 2002-04-23 Academia Sinica Treatments for spinal muscular atrophy
GB0219143D0 (en) * 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
EP1560931B1 (fr) * 2002-11-14 2011-07-27 Dharmacon, Inc. Arnsi fonctionnel et hyperfonctionnel
WO2004113867A2 (fr) 2003-06-16 2004-12-29 University Of Massachusetts Analyse d'exon
GB0425625D0 (en) * 2004-11-22 2004-12-22 Royal College Of Surgeons Ie Treatment of disease
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
PL3308788T3 (pl) 2005-06-23 2019-05-31 Biogen Ma Inc Kompozycje i sposoby modulacji splicingu smn2
WO2010091308A2 (fr) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Composés oligomères et procédés connexes
WO2010120820A1 (fr) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions et procédés pour moduler l'épissage de smn2
PT3449926T (pt) * 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito

Also Published As

Publication number Publication date
EP3308788A1 (fr) 2018-04-18
CY2017040I1 (el) 2018-04-04
ES2545223T3 (es) 2015-09-09
PL1910395T3 (pl) 2013-03-29
WO2007002390A3 (fr) 2007-11-15
US20150353929A1 (en) 2015-12-10
US8361977B2 (en) 2013-01-29
HK1181307A1 (en) 2013-11-08
EP1910395A2 (fr) 2008-04-16
DK1910395T3 (da) 2013-02-04
CY2017040I2 (el) 2018-04-04
EP3308788B1 (fr) 2018-10-24
LTC2548560I2 (lt) 2018-11-12
PT1910395E (pt) 2012-12-03
US20100216238A1 (en) 2010-08-26
US20180291376A1 (en) 2018-10-11
LUC00046I1 (fr) 2017-11-10
HUS1700042I1 (hu) 2017-12-28
EP2644700A1 (fr) 2013-10-02
DK2548560T3 (en) 2015-07-13
LT3308788T (lt) 2018-12-10
LTPA2017037I1 (lt) 2017-11-27
ES2397113T3 (es) 2013-03-04
PL2548560T3 (pl) 2015-11-30
SI2548560T1 (sl) 2015-12-31
US20130109091A1 (en) 2013-05-02
SI3308788T1 (sl) 2019-01-31
US8946183B2 (en) 2015-02-03
ES2702531T3 (es) 2019-03-01
EP3470072A1 (fr) 2019-04-17
EP2548560B1 (fr) 2015-06-03
EP2548560A1 (fr) 2013-01-23
WO2007002390A2 (fr) 2007-01-04
EP1910395A4 (fr) 2010-09-22
CY1121134T1 (el) 2019-12-11
DK3308788T3 (da) 2019-01-02
HK1247103B (en) 2019-09-06
US20170088835A1 (en) 2017-03-30
HUE027486T2 (en) 2016-09-28
PT3308788T (pt) 2019-01-30
EP1910395B1 (fr) 2012-10-17
PT2548560E (pt) 2015-09-22
US20200181613A1 (en) 2020-06-11
PL3308788T3 (pl) 2019-05-31

Similar Documents

Publication Publication Date Title
BE2023C529I2 (fr)
BE2021C524I2 (fr)
BE2021C519I2 (fr)
BE2020C531I2 (fr)
BE2020C511I2 (fr)
BE2020C508I2 (fr)
BE2020C004I2 (fr)
BE2019C521I2 (fr)
BE2019C503I2 (fr)
BE2018C037I2 (fr)
BE2018C035I2 (fr)
LUC00046I1 (fr)
BE2017C028I2 (fr)
BE2019C533I2 (fr)
BE2015C074I2 (fr)
BE2014C057I2 (fr)
BE2013C074I2 (fr)
BE2013C014I2 (fr)
BE2012C042I2 (fr)
BRPI0601358B8 (pt) Aplicador de clipe cirúrgico
BRPI0601402B8 (pt) Aplicador de grampos cirúrgicos
BR122017004709A2 (fr)
BRPI0609157A8 (fr)
BE2019C013I2 (fr)
BRPI0608519A2 (fr)