JP7506405B2 - 真核生物の遺伝子編集のためのレンチウイルスベースのベクターならびに関連システムおよび方法 - Google Patents

真核生物の遺伝子編集のためのレンチウイルスベースのベクターならびに関連システムおよび方法 Download PDF

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JP7506405B2
JP7506405B2 JP2020560827A JP2020560827A JP7506405B2 JP 7506405 B2 JP7506405 B2 JP 7506405B2 JP 2020560827 A JP2020560827 A JP 2020560827A JP 2020560827 A JP2020560827 A JP 2020560827A JP 7506405 B2 JP7506405 B2 JP 7506405B2
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バイソン ルー，
アンソニー アタラ，
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Wake Forest University Health Sciences
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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JP2020560827A 2018-05-01 2019-05-01 真核生物の遺伝子編集のためのレンチウイルスベースのベクターならびに関連システムおよび方法 Active JP7506405B2 (ja)

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Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3788155A1 (en) 2018-05-01 2021-03-10 Wake Forest University Health Sciences Lentiviral-based vectors and related systems and methods for eukaryotic gene editing
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
AU2019343045A1 (en) 2018-09-18 2021-05-13 Vnv Newco Inc. ARC-based capsids and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN110819707B (zh) * 2019-11-24 2023-07-18 中南大学湘雅医院 一种针对多种来源细胞样本中内部核糖体结合位点元件的高通量鉴定方法
WO2021142343A1 (en) * 2020-01-09 2021-07-15 The Regents Of The University Of California Targeting transfer rna for the suppression of nonsense mutations in messenger rna
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
WO2022109275A2 (en) * 2020-11-19 2022-05-27 Wake Forest University Health Sciences Vectors, systems and methods for eukaryotic gene editing
WO2022155239A1 (en) * 2021-01-12 2022-07-21 University Of Massachusetts Allele-specific inactivation of mutant htt via gene editing at coding region single nucleotide polymorphisms
US20240084330A1 (en) * 2021-01-28 2024-03-14 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
WO2022174004A1 (en) * 2021-02-12 2022-08-18 Wake Forest University Health Sciences Engineered extracellular vesicles and their uses
WO2022216536A1 (en) * 2021-04-06 2022-10-13 Animatus Biosciences, Inc. Selective expression of mrnas in cardiomyocytes without cardiac fibroblast stimulation
EP4341404A1 (en) * 2021-05-21 2024-03-27 Aarhus Universitet Lentivirus-derived nanoparticles comprising crispr/cas9 ribonucleoprotein complexes
JP7125727B1 (ja) 2021-09-07 2022-08-25 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法
EP4489772A4 (en) * 2022-03-08 2026-03-25 Univ Wake Forest Health Sciences EUKARYOTIC GENE EDITING COMPOSITIONS, SYSTEMS AND METHODS
CN116218906A (zh) * 2023-01-31 2023-06-06 安可来(重庆)生物医药科技有限公司 一种rna编辑器表达质粒、外泌体适配子融合表达质粒以及一种靶向基因rna编辑方法
WO2025242168A1 (en) * 2024-05-22 2025-11-27 Nanjing Legend Biotech Co., Ltd. Methods of making cells expressing an engineered receptor
CN118903442A (zh) * 2024-07-19 2024-11-08 上海佰炼生物医药科技有限公司 基于PP7 RNA茎环结构的高效VLP载体及其用于递送mRNA
CN119662735A (zh) * 2024-10-10 2025-03-21 上海佰炼生物医药科技有限公司 一种基于核定位信号和pp7 rna茎环结构的重组质粒及其载体系统和应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017194902A2 (fr) 2016-05-13 2017-11-16 Vectalys Particule pour l'encapsidation d'un système d'ingénierie du génome

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004225520A1 (en) 2003-03-25 2004-10-14 Stratagene DNA polymerase fusions and uses thereof
GB0526210D0 (en) * 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
US20120202276A1 (en) 2010-02-26 2012-08-09 Life Technologies Corporation Modified Proteins and Methods of Making and Using Same
WO2015002956A1 (en) 2013-07-01 2015-01-08 Ohio State Innovation Foundation Exosome delivery system
EP3080259B1 (en) * 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US9580727B1 (en) * 2015-08-07 2017-02-28 Caribou Biosciences, Inc. Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
US10624849B2 (en) 2015-09-28 2020-04-21 Northwestern University Targeted extracellular vesicles comprising membrane proteins with engineered glycosylation sites
WO2017059902A1 (en) * 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
CN105861485B (zh) * 2016-04-20 2021-08-17 上海伊丽萨生物科技有限公司 一种提高基因置换效率的方法
US11866697B2 (en) 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
KR102761791B1 (ko) 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
EP3788155A1 (en) 2018-05-01 2021-03-10 Wake Forest University Health Sciences Lentiviral-based vectors and related systems and methods for eukaryotic gene editing
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
AU2020466994A1 (en) 2019-09-17 2022-04-21 Rutgers, The State University Of New Jersey Highly efficient DNA base editors mediated by RNA-aptamer recruitment for targeted genome modification and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017194902A2 (fr) 2016-05-13 2017-11-16 Vectalys Particule pour l'encapsidation d'un système d'ingénierie du génome

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Biochemistry，2004年，Vol. 43, No. 14，p.4304-4312

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