CN109312308A - 通过使用核酸酶来进行人神经干细胞的基因组编辑 - Google Patents

通过使用核酸酶来进行人神经干细胞的基因组编辑 Download PDF

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Publication number
CN109312308A
CN109312308A CN201780036763.3A CN201780036763A CN109312308A CN 109312308 A CN109312308 A CN 109312308A CN 201780036763 A CN201780036763 A CN 201780036763A CN 109312308 A CN109312308 A CN 109312308A
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cell
stem cell
neural stem
dna
disease
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Chinese (zh)
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M·H·波蒂厄斯
E·J·基尔德贝克
D·P·德维尔
J·T·克拉克
A·冢本
N·内田
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Leland Stanford Junior University
Bright Ocens Silicon Valley Inc
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Leland Stanford Junior University
Bright Ocens Silicon Valley Inc
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CN201780036763.3A 2016-04-14 2017-04-13 通过使用核酸酶来进行人神经干细胞的基因组编辑 Pending CN109312308A (zh)

Applications Claiming Priority (3)

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US201662322652P 2016-04-14 2016-04-14
US62/322,652 2016-04-14
PCT/US2017/027504 WO2017180926A1 (en) 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases

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US (2) US20170298348A1 (https=)
EP (1) EP3443085B1 (https=)
JP (1) JP7197363B2 (https=)
CN (1) CN109312308A (https=)
AU (2) AU2017250683A1 (https=)
CA (1) CA3020181A1 (https=)
SG (1) SG11201808920RA (https=)
WO (1) WO2017180926A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110075319A (zh) * 2019-04-29 2019-08-02 白涛 一种抗遗传病生物导弹
CN111925998A (zh) * 2020-06-09 2020-11-13 广州再生医学与健康广东省实验室 模拟SARS-CoV-2感染的系统及其制备方法与应用
CN114369621A (zh) * 2021-12-07 2022-04-19 深圳市先康达生命科学有限公司 一种基因生物制剂及其制备方法和应用
CN115335512A (zh) * 2020-03-11 2022-11-11 瑞莫特治疗公司 用于在整个中枢神经系统中传播蛋白质的方法和材料

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したＣａｓ９蛋白質
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021163327A1 (en) * 2020-02-11 2021-08-19 University Of Virginia Patent Foundation Compositions and methods for treating and/or preventing ocular disorders
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR20230019156A (ko) * 2020-06-02 2023-02-07 카탈렌트 파마 솔루션즈, 엘엘씨 유전자 삽입을 위한 다수의 도크들이 있는 세포주
WO2022163770A1 (ja) * 2021-01-28 2022-08-04 国立研究開発法人理化学研究所 ゲノム編集ツールの評価方法

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130280222A1 (en) * 2012-04-18 2013-10-24 Board Of Regents Of The University Of Texas System Non-disruptive gene targeting
WO2015073867A1 (en) * 2013-11-15 2015-05-21 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Engineering neural stem cells using homologous recombination
CN105142669A (zh) * 2012-12-06 2015-12-09 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7037719B1 (en) 1999-02-12 2006-05-02 Stemcells California, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
US7217565B2 (en) 1999-02-12 2007-05-15 Stemcells California, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
US7105150B2 (en) 1999-02-12 2006-09-12 Stemcells California, Inc. In vivo screening methods using enriched neural stem cells
JP2006511206A (ja) 2002-08-27 2006-04-06 ステムセルズ， インコーポレイテッド 富化された中枢神経系幹細胞集団および前駆体細胞集団、ならびにこれらの集団を同定し、単離し、そして富化する方法
EP3241902B1 (en) * 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN105121648B (zh) * 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
US10117899B2 (en) * 2013-10-17 2018-11-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130280222A1 (en) * 2012-04-18 2013-10-24 Board Of Regents Of The University Of Texas System Non-disruptive gene targeting
CN105142669A (zh) * 2012-12-06 2015-12-09 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
WO2015073867A1 (en) * 2013-11-15 2015-05-21 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Engineering neural stem cells using homologous recombination
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ANGELO LOMBARDO等: "Site-specific integration and tailoring of cassette design for sustainable gene transfer", 《NATURE METHODS》 *
ELIAS T. ZAMBIDIS等: "Transcription Activator-Like Effector Nuclease (TALEN)-Mediated CLYBL Targeting Enables Enhanced Transgene Expression and One-Step Generation of Dual Reporter Human Induced Pluripotent Stem Cell (iPSC) and Neural Stem Cell (NSC) Lines", 《PLOS ONE》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110075319A (zh) * 2019-04-29 2019-08-02 白涛 一种抗遗传病生物导弹
CN115335512A (zh) * 2020-03-11 2022-11-11 瑞莫特治疗公司 用于在整个中枢神经系统中传播蛋白质的方法和材料
CN111925998A (zh) * 2020-06-09 2020-11-13 广州再生医学与健康广东省实验室 模拟SARS-CoV-2感染的系统及其制备方法与应用
CN114369621A (zh) * 2021-12-07 2022-04-19 深圳市先康达生命科学有限公司 一种基因生物制剂及其制备方法和应用
CN114369621B (zh) * 2021-12-07 2023-10-31 深圳市先康达生命科学有限公司 一种基因生物制剂及其制备方法和应用

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