[go: up one dir, main page]

PE20220256A1 - Sistemas de casx artificiales - Google Patents

Sistemas de casx artificiales

Info

Publication number
PE20220256A1
PE20220256A1 PE2021002050A PE2021002050A PE20220256A1 PE 20220256 A1 PE20220256 A1 PE 20220256A1 PE 2021002050 A PE2021002050 A PE 2021002050A PE 2021002050 A PE2021002050 A PE 2021002050A PE 20220256 A1 PE20220256 A1 PE 20220256A1
Authority
PE
Peru
Prior art keywords
casx
variant
artificial
systems
nag
Prior art date
Application number
PE2021002050A
Other languages
English (en)
Inventor
Benjamin Oakes
Sean Higgins
Hannah Spinner
Sarah Denny
Brett T Staahl
Kian Taylor
Katherine Baney
Isabel Colin
Maroof Adil
Original Assignee
Scribe 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 Scribe Therapeutics Inc filed Critical Scribe Therapeutics Inc
Publication of PE20220256A1 publication Critical patent/PE20220256A1/es

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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

En la presente se proporcionan sistemas de CasX y componentes de estos, incluidas proteinas CasX variantes y acidos nucleicos guia variantes (ANg). Las proteinas CasX variantes y ANg variantes de la divulgacion muestran por lo menos una caracteristica mejorada cuando se los compara con una proteina CasX de referencia o un ANg de referencia de la divulgacion. En algunas realizaciones, las variantes tienen una o mas funciones mejoradas del complejo de ribonucleoproteinas de CasX. Tambien se proveen metodos para producir y usar dichas variantes
PE2021002050A 2019-06-07 2020-06-05 Sistemas de casx artificiales PE20220256A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962858750P 2019-06-07 2019-06-07
US201962944892P 2019-12-06 2019-12-06
US202063030838P 2020-05-27 2020-05-27
PCT/US2020/036505 WO2020247882A1 (en) 2019-06-07 2020-06-05 Engineered casx systems

Publications (1)

Publication Number Publication Date
PE20220256A1 true PE20220256A1 (es) 2022-02-21

Family

ID=71899867

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021002050A PE20220256A1 (es) 2019-06-07 2020-06-05 Sistemas de casx artificiales

Country Status (17)

Country Link
US (5) US11560555B2 (es)
EP (1) EP3980533A1 (es)
JP (2) JP7744011B2 (es)
KR (1) KR20220032050A (es)
CN (1) CN114375334A (es)
AU (1) AU2020289591A1 (es)
BR (1) BR112021024288A2 (es)
CA (1) CA3142883A1 (es)
CL (1) CL2021003233A1 (es)
GB (1) GB2600274B (es)
IL (1) IL288738A (es)
MX (1) MX2021015058A (es)
PE (1) PE20220256A1 (es)
PH (1) PH12021553076A1 (es)
SG (1) SG11202113253SA (es)
TW (1) TW202113074A (es)
WO (1) WO2020247882A1 (es)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020041456A1 (en) * 2018-08-22 2020-02-27 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
WO2021050593A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
CA3159316A1 (en) 2019-12-06 2021-06-10 Benjamin OAKES Compositions and methods for the targeting of rhodopsin
EP4087930A1 (en) * 2020-01-10 2022-11-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN116096885A (zh) 2020-03-18 2023-05-09 斯克里贝治疗公司 用于靶向C9orf72的组合物和方法
EP4256045A4 (en) * 2020-12-02 2025-01-08 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
EP4256054A1 (en) 2020-12-03 2023-10-11 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022120089A1 (en) 2020-12-03 2022-06-09 Scribe Therapeutics Inc. Compositions and methods for the targeting of ptbp1
EP4259791A1 (en) 2020-12-09 2023-10-18 Scribe Therapeutics Inc. Aav vectors for gene editing
TW202302844A (zh) * 2021-03-09 2023-01-16 美商阿伯生物技術公司 包含變異多肽之組合物及其用途
US20240102007A1 (en) 2021-06-01 2024-03-28 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
WO2023049742A2 (en) * 2021-09-21 2023-03-30 Scribe Therapeutics Inc. Engineered casx repressor systems
IL311611A (en) 2021-09-23 2024-05-01 Scribe Therapeutics Inc Self-inactivating gene-editing vectors
CN116497002A (zh) * 2022-01-19 2023-07-28 中国科学院动物研究所 工程化的CasX核酸酶、效应蛋白及其用途
AU2023278164A1 (en) 2022-06-02 2024-12-05 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240076A1 (en) * 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
WO2023240157A2 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
EP4536286A1 (en) 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024081711A2 (en) * 2022-10-11 2024-04-18 The Broad Institute, Inc. Reprogramable tnpb polypeptides and use thereof
WO2024173645A1 (en) 2023-02-15 2024-08-22 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
EP4680287A1 (en) 2023-03-15 2026-01-21 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
TW202444914A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 抑制子融合蛋白系統
WO2024206620A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Messenger rna encoding casx
AU2024242151A1 (en) 2023-03-29 2025-09-25 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
TW202444906A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向pcsk9之組合物及方法
CN116926043B (zh) * 2023-04-28 2024-09-06 新乡医学院第三附属医院 一种dCasX蛋白核酸酶、重组载体及其应用
WO2024228975A2 (en) * 2023-05-02 2024-11-07 Inari Agriculture Technology, Inc. Ribozyme sequences for processing rna
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
CN121127501A (zh) * 2023-06-16 2025-12-12 株式会社维赛基因科技 锌指蛋白、锌指核酸酶、载体和应用它们的基因组编辑方法
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
CN117296799B (zh) * 2023-11-28 2024-02-02 四川省医学科学院·四川省人民医院 一种视网膜色素变性疾病模型的构建方法及其应用
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
CN120424908B (zh) * 2025-07-10 2025-09-12 南京农业大学 一种基于LAMP与CRISPR/Cas的检测方法和应用

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
EP1179340A3 (en) 1994-09-30 2003-05-07 INEX Pharmaceutical Corp. Compositions for the introduction of polyanionic materials into cells
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
CA2597724A1 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
JP2012508017A (ja) 2008-11-07 2012-04-05 ファブラス エルエルシー 抗ｄｌｌ４抗体及びその使用
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
PT3241902T (pt) 2012-05-25 2018-05-28 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP3160938B1 (en) 2014-06-25 2020-09-16 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016118697A1 (en) 2015-01-21 2016-07-28 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016285852B2 (en) 2015-06-29 2020-12-17 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3365441A1 (en) 2015-10-22 2018-08-29 The Broad Institute Inc. Type vi-b crispr enzymes and systems
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US11118194B2 (en) 2015-12-18 2021-09-14 The Regents Of The University Of California Modified site-directed modifying polypeptides and methods of use thereof
US9896696B2 (en) 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
WO2017212008A1 (en) 2016-06-09 2017-12-14 Curevac Ag Hybrid carriers for nucleic acid cargo
EP3468609A1 (en) 2016-06-09 2019-04-17 CureVac AG Cationic carriers for nucleic acid delivery
US10017760B2 (en) 2016-06-24 2018-07-10 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CN110023494A (zh) 2016-09-30 2019-07-16 加利福尼亚大学董事会 Rna指导的核酸修饰酶及其使用方法
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
JP7308380B2 (ja) 2017-01-10 2023-07-14 クリスティアーナ ケア ジーン エディティング インスティテュート，インコーポレイテッド 遺伝子編集技術を用いたインビトロ部位特異的変異導入のための方法
WO2018152418A1 (en) * 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
EP3601576A1 (en) 2017-03-24 2020-02-05 CureVac AG Nucleic acids encoding crispr-associated proteins and uses thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
EP3701013A4 (en) 2017-10-25 2021-08-04 Monsanto Technology LLC TARGETED ENDONUCLEASE ACTIVITY OF RNA-GUIDED ENDONUCLEASE CASX IN EUKARYONTS
US10253365B1 (en) 2017-11-22 2019-04-09 The Regents Of The University Of California Type V CRISPR/Cas effector proteins for cleaving ssDNAs and detecting target DNAs
US20200224160A1 (en) 2018-02-27 2020-07-16 Sorrento Therapeutics, Inc. Process for dna integration using rna-guided endonucleases
CA3091912A1 (en) 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
WO2019213273A1 (en) 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
EP3818167A4 (en) 2018-07-05 2022-04-13 The Regents of The University of California COMPOSITIONS AND METHODS FOR DELIVERING RNA TO A CELL
US20210284981A1 (en) 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
WO2020041456A1 (en) * 2018-08-22 2020-02-27 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
WO2021007177A1 (en) 2019-07-08 2021-01-14 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
JP7765377B2 (ja) 2019-08-02 2025-11-06 モンサント テクノロジー エルエルシー Ｈｕｈエンドヌクレアーゼを用いた標的化ゲノム修飾を促進するための方法及び組成物
WO2021050593A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
CA3153700A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
WO2021084533A1 (en) 2019-10-28 2021-05-06 Targetgene Biotechnologies Ltd Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation
AU2020398658A1 (en) 2019-12-06 2022-07-07 Scribe Therapeutics Inc. Particle delivery systems
CA3159316A1 (en) 2019-12-06 2021-06-10 Benjamin OAKES Compositions and methods for the targeting of rhodopsin
EP4069846A1 (en) 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
EP4087930A1 (en) 2020-01-10 2022-11-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN116096885A (zh) 2020-03-18 2023-05-09 斯克里贝治疗公司 用于靶向C9orf72的组合物和方法
EP4256054A1 (en) 2020-12-03 2023-10-11 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
WO2023049742A2 (en) 2021-09-21 2023-03-30 Scribe Therapeutics Inc. Engineered casx repressor systems
IL311611A (en) 2021-09-23 2024-05-01 Scribe Therapeutics Inc Self-inactivating gene-editing vectors
AU2023278164A1 (en) 2022-06-02 2024-12-05 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems

Also Published As

Publication number Publication date
GB2600274B (en) 2024-11-27
GB2600274A (en) 2022-04-27
US20250043262A1 (en) 2025-02-06
IL288738A (en) 2022-02-01
EP3980533A1 (en) 2022-04-13
US12084692B2 (en) 2024-09-10
US20250136962A1 (en) 2025-05-01
PH12021553076A1 (en) 2023-08-14
TW202113074A (zh) 2021-04-01
JP7744011B2 (ja) 2025-09-25
US20230124880A1 (en) 2023-04-20
CA3142883A1 (en) 2020-12-10
MX2021015058A (es) 2022-04-06
CL2021003233A1 (es) 2022-08-12
BR112021024288A2 (pt) 2022-02-15
KR20220032050A (ko) 2022-03-15
JP2022534809A (ja) 2022-08-03
AU2020289591A1 (en) 2021-12-23
JP2025183286A (ja) 2025-12-16
US20220081681A1 (en) 2022-03-17
WO2020247882A1 (en) 2020-12-10
CN114375334A (zh) 2022-04-19
SG11202113253SA (en) 2021-12-30
US11560555B2 (en) 2023-01-24
US20220220508A1 (en) 2022-07-14

Similar Documents

Publication Publication Date Title
PE20220256A1 (es) Sistemas de casx artificiales
CL2020002161A1 (es) Composiciones y métodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano.
MX2022006760A (es) Composiciones de proteina anti-vegf y metodos para producir la misma.
MX2020011257A (es) Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos.
CO2019000395A2 (es) Genes de mini–distrofina optimizados y casetes de expresión y su uso
CL2023001793A1 (es) Anticuerpos anti-componente de complemento y métodos de uso
EP3755331A4 (en) AGONISTS OF THE STIMULATOR OF INTERFERON GENES (STING)
CO2020000774A2 (es) Rellenos de tejidos basados en ácido hialurónico de seda y métodos de utilizar los mismos
MX2022008073A (es) Anticuerpos dirigidos a los huesos.
MX2020002921A (es) Anticuerpos para claudin6 y métodos para tratar el cáncer.
AR110114A1 (es) ANTICUERPOS CONTRA b-AMILOIDE PIROGLUTAMATO Y SUS USOS
AR113490A1 (es) CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
PE20190126A1 (es) Proteinas de union y metodos de uso de las mismas
AR106144A1 (es) Un conjugado de proteína que comprende polipéptidos fisiológicos y una región fc inmunoglobulina
EP3849434A4 (en) MACHINE VISION TECHNIQUES
MX2018004531A (es) Proteinas de union a antigeno que activan el receptor de leptina.
CY1121187T1 (el) Ενα εκχυλισμα απο indigo naturalis και μια διεργασια για την παρασκευη εκεινου
EP3902556A4 (en) IN VIVO MANIPULATED CEREBLON PROTEIN
CO2020016105A2 (es) Proteínas inmunodominantes y fragmentos en esclerosis múltiple
CY1122644T1 (el) Πρωτεϊνες συντηξης tatk-cdkl5, συνθεσεις, φαρμακοτεχνικες μορφες και η χρηση αυτων
EP3893658C0 (en) LOW SODIUM PROTEIN ISOLATE
MX2020011529A (es) Un metodo para optimizar la expresion de anticuerpos.
EA201990864A1 (ru) Конструкции для экспрессии фратаксина
DK3737402T5 (da) Modificeret protein
MX2019004371A (es) Anticuerpos que se unen a la proteina de envoltura del virus zika y usos de los mismos.