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)
Inventor
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)

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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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EP (1) EP3443085B1 (enExample)
JP (1) JP7197363B2 (enExample)
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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
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
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
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 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
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
WO2020214842A1 (en) 2019-04-17 2020-10-22 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
US20230355553A1 (en) * 2020-02-11 2023-11-09 University Of Virginia Patent Foundation Compositions and methods for treating and/or preventing ocular disorders
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
AU2021283272A1 (en) * 2020-06-02 2023-01-19 Catalent Pharma Solutions, Llc Cell lines with multiple docks for gene insertion
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
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
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
US7381561B2 (en) 2002-08-27 2008-06-03 Stemcells California, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
PL4289948T3 (pl) * 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
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
CA2926078C (en) * 2013-10-17 2021-11-16 Sangamo Biosciences, 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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EP3443085B1 (en) 2022-09-14
AU2023208065A1 (en) 2023-08-17
US20170298348A1 (en) 2017-10-19
JP2019511240A (ja) 2019-04-25
EP3443085A4 (en) 2019-09-25
JP7197363B2 (ja) 2022-12-27
CA3020181A1 (en) 2017-10-19
US20230250423A1 (en) 2023-08-10

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