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)

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 (enExample)
EP (1) EP3443085B1 (enExample)
JP (1) JP7197363B2 (enExample)
CN (1) CN109312308A (enExample)
AU (2) AU2017250683A1 (enExample)
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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

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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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CA3033327A1 (en) 2016-08-09 2018-02-15 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
JP7588390B2 (ja) 2016-10-14 2024-11-22 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 核酸塩基エディターのａａｖ送達
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
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
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
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
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 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
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US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 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
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CA3180217A1 (en) * 2020-06-02 2021-12-09 Gregory T. Bleck Nucleic acid constructs for protein manufacture
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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
JP7197363B2 (ja) 2022-12-27
EP3443085A1 (en) 2019-02-20
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EP3443085A4 (en) 2019-09-25
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AU2023208065A1 (en) 2023-08-17
US20170298348A1 (en) 2017-10-19

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