JP2023546597A - 部位特異的標的化エレメントによるプログラム可能な付加(paste)を使用した部位特異的遺伝子操作のためのシステム、方法及び組成物 - Google Patents

部位特異的標的化エレメントによるプログラム可能な付加(paste)を使用した部位特異的遺伝子操作のためのシステム、方法及び組成物 Download PDF

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JP2023546597A
JP2023546597A JP2023524355A JP2023524355A JP2023546597A JP 2023546597 A JP2023546597 A JP 2023546597A JP 2023524355 A JP2023524355 A JP 2023524355A JP 2023524355 A JP2023524355 A JP 2023524355A JP 2023546597 A JP2023546597 A JP 2023546597A
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dna
site
cell
integration
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アブディエ,オマー
グーテンバーグ,ジョナサン
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Massachusetts Institute of Technology
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JP2023524355A 2020-10-21 2021-10-21 部位特異的標的化エレメントによるプログラム可能な付加(paste)を使用した部位特異的遺伝子操作のためのシステム、方法及び組成物 Pending JP2023546597A (ja)

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US202063094803P 2020-10-21 2020-10-21
US63/094,803 2020-10-21
US202163222550P 2021-07-16 2021-07-16
US63/222,550 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)

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JP2023546597A true JP2023546597A (ja) 2023-11-06
JP2023546597A5 JP2023546597A5 (enExample) 2024-10-15

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CN (1) CN116419975A (enExample)
AU (2) AU2021364781B2 (enExample)
CA (1) CA3196116A1 (enExample)
IL (1) IL301368A (enExample)
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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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016537008A (ja) * 2013-09-06 2016-12-01 プレジデント アンド フェローズ オブ ハーバード カレッジ Cas9バリアントおよびその使用
WO2020191245A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

Family Cites Families (30)

* 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
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
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016537008A (ja) * 2013-09-06 2016-12-01 プレジデント アンド フェローズ オブ ハーバード カレッジ Cas9バリアントおよびその使用
WO2020191245A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020191234A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

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