[go: up one dir, main page]

PE20231178A1 - Sistemas crispr tipo v clase 2 disenados por ingenieria - Google Patents

Sistemas crispr tipo v clase 2 disenados por ingenieria

Info

Publication number
PE20231178A1
PE20231178A1 PE2023001812A PE2023001812A PE20231178A1 PE 20231178 A1 PE20231178 A1 PE 20231178A1 PE 2023001812 A PE2023001812 A PE 2023001812A PE 2023001812 A PE2023001812 A PE 2023001812A PE 20231178 A1 PE20231178 A1 PE 20231178A1
Authority
PE
Peru
Prior art keywords
type
crispr systems
class
designed class
engineering designed
Prior art date
Application number
PE2023001812A
Other languages
English (en)
Inventor
Sean Higgins
Isabel Colin
Sarah Denny
Brett T Staahl
Benjamin Oakes
Angus Sidore
Suraj Makhija
Gayathri Vijayakumar
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 PE20231178A1 publication Critical patent/PE20231178A1/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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
    • C12N15/11—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
    • C12N15/63—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
    • 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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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
    • C12N2320/50—Methods for regulating/modulating their 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
    • 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
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

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

Abstract

En la presente descripcion se proporcionan nucleasas tipo V clase 2, y ARN guias disenados por ingenieria utiles para la edicion de acidos nucleicos diana. Tambien se proporcionan metodos para elaborar y usar tales variantes para modificar acidos nucleicos
PE2023001812A 2020-12-03 2021-12-02 Sistemas crispr tipo v clase 2 disenados por ingenieria PE20231178A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063121196P 2020-12-03 2020-12-03
US202163162346P 2021-03-17 2021-03-17
US202163208855P 2021-06-09 2021-06-09
PCT/US2021/061673 WO2022120095A1 (en) 2020-12-03 2021-12-02 Engineered class 2 type v crispr systems

Publications (1)

Publication Number Publication Date
PE20231178A1 true PE20231178A1 (es) 2023-08-01

Family

ID=80050633

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2023001812A PE20231178A1 (es) 2020-12-03 2021-12-02 Sistemas crispr tipo v clase 2 disenados por ingenieria

Country Status (12)

Country Link
US (4) US20240026385A1 (es)
EP (1) EP4256054A1 (es)
JP (1) JP2023552374A (es)
KR (1) KR20230128289A (es)
AU (1) AU2021392719A1 (es)
CA (1) CA3201258A1 (es)
CL (2) CL2023001594A1 (es)
GB (1) GB2616584A (es)
MX (1) MX2023006566A (es)
PE (1) PE20231178A1 (es)
TW (1) TW202237836A (es)
WO (1) WO2022120095A1 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2600274B (en) 2019-06-07 2024-11-27 Scribe Therapeutics Inc Engineered CasX systems
EP4069845A1 (en) 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
TW202237836A (zh) 2020-12-03 2022-10-01 美商斯奎柏治療公司 工程化之第2類第v型crispr系統
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
TW202320864A (zh) 2021-09-21 2023-06-01 美商斯奎柏治療公司 經工程改造之ｃａｓｘ抑制子系統
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
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
AU2023283464A1 (en) 2022-06-07 2024-12-05 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240157A2 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
WO2023240162A1 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing
CN116676291B (zh) * 2022-08-22 2024-02-27 华中农业大学 核酸内切酶Genie scissor及其介导的基因编辑系统
TW202444910A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 編碼casx之信使rna
WO2024206565A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Repressor fusion protein systems
TW202444921A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向lpa之組合物及方法
WO2024206555A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
JP7560818B1 (ja) 2023-08-25 2024-10-03 株式会社キュライオ エンジニアリングされたタンパク質

Family Cites Families (69)

* 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
US5785992A (en) 1994-09-30 1998-07-28 Inex Pharmaceuticals Corp. Compositions for the introduction of polyanionic materials into cells
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation 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
ATE537263T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Kationische lipide und verwendungsverfahren
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
CA3044134A1 (en) 2008-01-02 2009-07-09 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
JP5882058B2 (ja) 2008-11-07 2016-03-09 ファブラス エルエルシー 組合せ抗体ライブラリー及びその使用
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
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
LT2800811T (lt) 2012-05-25 2017-09-11 The Regents Of The University Of California Būdai ir kompozicijos, skirti tikslinės dnr modifikavimui, panaudojant adresuotą rnr, ir transkripcijos moduliavimui, panaudojant adresuotą rnr
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP3313829B1 (en) 2015-06-29 2024-04-10 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
KR102761827B1 (ko) 2015-10-22 2025-02-03 더 브로드 인스티튜트, 인코퍼레이티드 타입 vi-b crispr 효소 및 시스템
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY
EP3390624A4 (en) 2015-12-18 2019-07-10 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
WO2017212007A1 (en) 2016-06-09 2017-12-14 Curevac Ag Cationic carriers for nucleic acid delivery
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会（法人团体） 用于生成条形码化组合文库的方法
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
EA201990861A1 (ru) 2016-09-30 2019-09-30 Те Риджентс Оф Те Юниверсити Оф Калифорния Рнк-направляемые модифицирующие нуклеиновые кислоты ферменты и способы их применения
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
CA3048501A1 (en) 2017-01-10 2018-07-19 Christiana Care Health Services, Inc. Methods for in vitro site-directed mutagenesis using gene editing technologies
US20190367924A1 (en) 2017-02-17 2019-12-05 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
WO2019084148A1 (en) * 2017-10-25 2019-05-02 Monsanto Technology Llc TARGETED RNA GUIDED ENDONUCLEASE ENDONUCLEASE ACTIVITY IN EUKARYOTES
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
CA3091912A1 (en) 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
US20200224160A1 (en) 2018-02-27 2020-07-16 Sorrento Therapeutics, Inc. Process for dna integration using rna-guided endonucleases
US20210115420A1 (en) 2018-05-01 2021-04-22 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
US20210139892A1 (en) 2018-07-05 2021-05-13 The Regents Of The University Of California Compositions and methods for delivery of 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
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California Variant type v crispr/cas effector polypeptides and methods of use thereof
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
GB2600274B (en) * 2019-06-07 2024-11-27 Scribe Therapeutics Inc Engineered CasX systems
US20220315914A1 (en) 2019-07-08 2022-10-06 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
WO2021025999A1 (en) 2019-08-02 2021-02-11 Monsanto Technology Llc Methods and compositions to promote targeted genome modifications using huh endonucleases
WO2021050593A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
CN114729368A (zh) 2019-09-09 2022-07-08 斯克里贝治疗公司 用于免疫疗法的组合物和方法
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
EP4069845A1 (en) 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
KR20220113442A (ko) 2019-12-06 2022-08-12 스크라이브 테라퓨틱스 인크. 입자 전달 시스템
WO2021113769A1 (en) 2019-12-07 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
WO2021142342A1 (en) 2020-01-10 2021-07-15 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
MX2022011460A (es) * 2020-03-18 2022-12-15 Scribe Therapeutics Inc Composiciones y métodos para el direccionamiento de c9orf72.
US20230407276A1 (en) 2020-12-02 2023-12-21 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
TW202237836A (zh) 2020-12-03 2022-10-01 美商斯奎柏治療公司 工程化之第2類第v型crispr系統
EP4314297A1 (en) 2021-03-26 2024-02-07 Specific Biologics Inc. Targeting oncogentic mutations with dual-cleaving endonuclease
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
TW202320864A (zh) 2021-09-21 2023-06-01 美商斯奎柏治療公司 經工程改造之ｃａｓｘ抑制子系統
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
EP4256054A1 (en) 2023-10-11
CL2023001594A1 (es) 2024-01-05
US12163125B2 (en) 2024-12-10
CA3201258A1 (en) 2022-06-09
JP2023552374A (ja) 2023-12-15
TW202237836A (zh) 2022-10-01
WO2022120095A1 (en) 2022-06-09
US20230054437A1 (en) 2023-02-23
CL2024001727A1 (es) 2024-10-18
GB202309876D0 (en) 2023-08-16
MX2023006566A (es) 2023-08-07
US20240376462A1 (en) 2024-11-14
KR20230128289A (ko) 2023-09-04
AU2021392719A1 (en) 2023-06-22
US20240026385A1 (en) 2024-01-25
GB2616584A (en) 2023-09-13
US20250011756A1 (en) 2025-01-09
AU2021392719A9 (en) 2024-10-03

Similar Documents

Publication Publication Date Title
PE20231178A1 (es) Sistemas crispr tipo v clase 2 disenados por ingenieria
CO2022014598A2 (es) Composiciones y métodos para el direccionamiento de c9orf72
MX2020006072A (es) Métodos y composiciones relacionados con cpf1 para la edición genica.
MX2024006956A (es) Inserciones de adn no virales orientadas.
MX2024007924A (es) Edicion genomica multiplexada.
CY1122347T1 (el) 3' utr αλληλουχιες για τη σταθεροποιηση toy rna
MX2020011939A (es) Nuevas enzimas y sistemas crispr.
AR098300A1 (es) Locus óptimos de la soja
BR112021026220A2 (pt) Edição aprimorada do genoma por reparo dependente de homologia
MX2019011272A (es) Composiciones y metodos para inmunooncologia.
NZ738689A (en) Engineered crispr-cas9 compositions and methods of use
MX2018012460A (es) Composiciones de conjugados de acido nucleico dirigido.
BR112017007770A2 (pt) cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
BR112018010429A2 (pt) ácidos nucleicos que se direcionam a ácidos nucleicos engenheirados
MX382223B (es) Regulacion de expresion genica mediada por nucleasa.
MX2023009345A (es) Compuestos, composiciones y metodos de uso de estos.
BR112021010781A2 (pt) Silenciamento genético por meio de edição de genoma
BR112022006260A2 (pt) Nuclease mad7 em plantas e expansão de sua capacidade de reconhecimento
EA201070229A1 (ru) Нуклеотидные последовательности msca1, влияющие на мужскую фертильность растений, и способ их применения
WO2023235818A3 (en) Engineered class 2 type v crispr systems
CO2023007900A2 (es) Composiciones y métodos para el direccionamiento de bcl11a
BR112022008468A2 (pt) Método para modificação direcionada de uma sequência de genoma de planta
BR112019005605A2 (pt) métodos para modificar o genoma (nuclear) de uma célula vegetal e um gene epsps endógeno ou para produzir uma célula vegetal ou para testar a eficiência de edição de genoma, bactéria, e, vetor
CL2022001481A1 (es) Composiciones de ácidos nucleicos
BR112021007503A2 (pt) Proteína de fusão, molécula de ácido nucleico, método para edição de material genético, e, sistemas para edição de material genético e para distribuição de componentes de edição de genoma