IL301368A - Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE) - Google Patents

Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE)

Info

Publication number
IL301368A
IL301368A IL301368A IL30136823A IL301368A IL 301368 A IL301368 A IL 301368A IL 301368 A IL301368 A IL 301368A IL 30136823 A IL30136823 A IL 30136823A IL 301368 A IL301368 A IL 301368A
Authority
IL
Israel
Prior art keywords
integration
site
cell
seq
sequence
Prior art date
Application number
IL301368A
Other languages
English (en)
Hebrew (he)
Inventor
Omar Abudayyeh
Jonathan Gootenberg
Original Assignee
Massachusetts Inst Technology
Omar Abudayyeh
Jonathan Gootenberg
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 Massachusetts Inst Technology, Omar Abudayyeh, Jonathan Gootenberg filed Critical Massachusetts Inst Technology
Publication of IL301368A publication Critical patent/IL301368A/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • C12N9/1276RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07Nucleotidyltransferases (2.7.7)
    • C12Y207/07049RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
IL301368A 2020-10-21 2021-10-21 Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE) IL301368A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063094803P 2020-10-21 2020-10-21
US202163222550P 2021-07-16 2021-07-16
PCT/US2021/056006 WO2022087235A1 (en) 2020-10-21 2021-10-21 Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)

Publications (1)

Publication Number Publication Date
IL301368A true IL301368A (en) 2023-05-01

Family

ID=78821040

Family Applications (1)

Application Number Title Priority Date Filing Date
IL301368A IL301368A (en) 2020-10-21 2021-10-21 Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE)

Country Status (10)

Country Link
US (7) US20220145293A1 (enExample)
EP (1) EP4232583A1 (enExample)
JP (1) JP2023546597A (enExample)
KR (1) KR20230091894A (enExample)
CN (1) CN116419975A (enExample)
AU (2) AU2021364781B2 (enExample)
CA (1) CA3196116A1 (enExample)
IL (1) IL301368A (enExample)
MX (1) MX2023004383A (enExample)
WO (1) WO2022087235A1 (enExample)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
KR20220097875A (ko) 2019-09-03 2022-07-08 마이얼로이드 테라퓨틱스, 인크. 게놈 통합을 위한 방법 및 조성물
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN116670154A (zh) 2020-07-24 2023-08-29 总医院公司 增强的病毒样颗粒及使用其递送至细胞的方法
AU2021364781B2 (en) 2020-10-21 2025-10-09 Massachusetts Institute Of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
GB202113933D0 (en) * 2021-09-29 2021-11-10 Genome Res Ltd Methods for gene editing
WO2023076898A1 (en) * 2021-10-25 2023-05-04 The Broad Institute, Inc. Methods and compositions for editing a genome with prime editing and a recombinase
WO2023077148A1 (en) * 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
US20230287441A1 (en) * 2021-12-17 2023-09-14 Massachusetts Institute Of Technology Programmable insertion approaches via reverse transcriptase recruitment
EP4452335A1 (en) 2021-12-22 2024-10-30 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
EP4493706A4 (en) * 2022-03-14 2026-03-18 Univ California INTEGRATION OF LARGE NUCLEIC ACIDS INTO GENOMES
WO2023205744A1 (en) * 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
EP4511486A1 (en) 2022-04-20 2025-02-26 Massachusetts Institute Of Technology Site specific genetic engineering utilizing trans-template rnas
WO2023205710A1 (en) 2022-04-20 2023-10-26 Massachusetts Institute Of Technology Programmable gene editing using guide rna pair
WO2023215831A1 (en) * 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) * 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
CN120344660A (zh) 2022-07-18 2025-07-18 雷纳嘉德医疗管理公司 基因编辑组分、系统和使用方法
WO2024020587A2 (en) * 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
CN116004586B (zh) * 2022-12-01 2025-08-26 上海药明生物医药有限公司 通过优化提高Bxb1酶的重组效率的方法
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
IL321929A (en) * 2023-01-05 2025-09-01 Prime Medicine Inc Genome editors and methods of use
WO2024168147A2 (en) * 2023-02-09 2024-08-15 The Broad Institute, Inc. Evolved recombinases for editing a genome in combination with prime editing
CN118546903A (zh) * 2023-02-24 2024-08-27 尧唐(上海)生物科技有限公司 能介导大片段dna整合到哺乳动物基因组的酶及其应用
WO2024220407A2 (en) * 2023-04-17 2024-10-24 Massachusetts Institute Of Technology Systems, methods, and compositions for cystic fibrosis gene editing
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024238595A2 (en) * 2023-05-16 2024-11-21 Sarepta Therapeutics, Inc. Fusion proteins comprising cas enzymes and dna-binding proteins
MX2025013657A (es) * 2023-05-16 2026-02-03 Prime Medicine Inc Composiciones y métodos de uso de edición del genoma con el gen b2m como objetivo
EP4731757A2 (en) 2023-06-26 2026-04-29 University of Hawaii Evolved integrases and methods of using the same for genome editing
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025147545A1 (en) 2024-01-03 2025-07-10 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids and related methods and uses
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
CN117720672B (zh) * 2024-02-07 2024-04-30 深锐(天津)生物医学有限公司 先导编辑系统及其应用
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
WO2025193915A1 (en) 2024-03-14 2025-09-18 Massachusetts Institute Of Technology High efficiency integrases for gene editing
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025226730A1 (en) * 2024-04-22 2025-10-30 Transit Therapeutics, Inc. Revision of genetic material using direct replacement editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
EP4703469A1 (en) 2024-08-26 2026-03-04 Charité - Universitätsmedizin Berlin Körperschaft des öffentlichen Rechts Fusion proteins, methods and compositions for efficient serine integrase-mediated gene transfer in human cells
WO2026046974A1 (en) 2024-08-26 2026-03-05 Charité - Universitätsmedizin Berlin Körperschaft des öffentlichen Rechts Fusion proteins, methods and compositions for efficient serine integrase-mediated gene transfer in human cells

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
SG11201507378UA (en) 2013-03-14 2015-10-29 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
US20140349400A1 (en) * 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
JP2017518082A (ja) 2014-06-17 2017-07-06 ポセイダ セラピューティクス, インコーポレイテッド ゲノム中の特異的遺伝子座にタンパク質を指向させるための方法およびその使用
US10059940B2 (en) 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2017151719A1 (en) 2016-03-01 2017-09-08 University Of Florida Research Foundation, Incorporated Molecular cell diary system
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018049168A1 (en) 2016-09-09 2018-03-15 The Board Of Trustees Of The Leland Stanford Junior University High-throughput precision genome editing
CN110291434B (zh) 2016-09-12 2021-11-05 麦斯卓有限公司 Mems致动系统和方法
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
CN111801417B (zh) 2017-12-14 2024-10-29 克里斯珀医疗股份公司 新的rna-可编程的内切核酸酶系统及其在基因组编辑和其他应用中的用途
CN113286880A (zh) 2018-08-28 2021-08-20 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
EP3942043A2 (en) * 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US10837021B1 (en) 2019-06-06 2020-11-17 Inscripta, Inc. Curing for recursive nucleic acid-guided cell editing
KR20220097875A (ko) 2019-09-03 2022-07-08 마이얼로이드 테라퓨틱스, 인크. 게놈 통합을 위한 방법 및 조성물
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4085141A4 (en) * 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING REVERSE TRANSCRIPTASE FOR FULLY ACTIVE CRISPR COMPLEXES
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia Therapeutics, Inc. Methods and compositions for directed genome editing
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2022060749A1 (en) * 2020-09-15 2022-03-24 Inscripta, Inc. Crispr editing to embed nucleic acid landing pads into genomes of live cells
JP2023543803A (ja) 2020-09-24 2023-10-18 ザ ブロード インスティテュート,インコーポレーテッド プライム編集ガイドrna、その組成物、及びその使用方法
AU2021364781B2 (en) 2020-10-21 2025-10-09 Massachusetts Institute Of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
EP4240840A4 (en) 2020-11-05 2025-01-01 University of Washington PRECISE GENOME DELETION AND REPLACEMENT METHOD BASED ON PRIMARY EDITING

Also Published As

Publication number Publication date
CA3196116A1 (en) 2022-04-28
AU2025287334A1 (en) 2026-01-22
US20240067961A1 (en) 2024-02-29
AU2025287334B2 (en) 2026-03-26
US11834658B2 (en) 2023-12-05
US20240076662A1 (en) 2024-03-07
US20250092390A1 (en) 2025-03-20
US20220145293A1 (en) 2022-05-12
MX2023004383A (es) 2023-05-04
US11952571B2 (en) 2024-04-09
US11572556B2 (en) 2023-02-07
US20230279391A1 (en) 2023-09-07
AU2021364781A1 (en) 2023-06-01
AU2021364781B2 (en) 2025-10-09
US20230135673A1 (en) 2023-05-04
KR20230091894A (ko) 2023-06-23
US12195733B2 (en) 2025-01-14
CN116419975A (zh) 2023-07-11
WO2022087235A1 (en) 2022-04-28
US20220154224A1 (en) 2022-05-19
JP2023546597A (ja) 2023-11-06
EP4232583A1 (en) 2023-08-30
US11827881B2 (en) 2023-11-28

Similar Documents

Publication Publication Date Title
US12195733B2 (en) Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
US11124796B2 (en) Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for modeling competition of multiple cancer mutations in vivo
JP2024157566A (ja) 最適化機能CRISPR-Cas系による配列操作のための系、方法および組成物
JP2025118829A (ja) ゲノム編集分子の細胞内送達のためのペプチドおよびナノ粒子
CN107513538A (zh) 基因敲除方法
CN106103705A (zh) 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
US20240309348A1 (en) Systems, methods, and compositions comprising miniature crispr nucleases for gene editing and programmable gene activation and inhibition
WO2023081756A1 (en) Precise genome editing using retrons
JP7843231B2 (ja) 遺伝子発現を増強させる哺乳動物配列への標的組込み
Jin et al. Optimizing homology-directed repair for gene editing: the potential of single-stranded DNA donors
JP7109009B2 (ja) 遺伝子ノックアウト方法
Nan et al. Ligase-mediated programmable genomic integration (L-PGI): an efficient site-specific gene editing system that overcomes the limitations of reverse transcriptase-based editing systems
US20230287441A1 (en) Programmable insertion approaches via reverse transcriptase recruitment
AU2023329386A1 (en) Programmable rna writing using crispr effectors and trans-splicing templates
WO2020117992A9 (en) Improved vector systems for cas protein and sgrna delivery, and uses therefor
WO2024192274A2 (en) On cas template synthesis (ocats) systems and uses thereof
EP4662324A2 (en) Integrase variants for gene insertion in human cell
Martin Improved Base Editing Technologies With Novel Editors and Assays
Ochmann Development and characterization of a next generation genome engineering tool for gene therapy