JP2019511240A5 - - Google Patents

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JP2019511240A5
JP2019511240A5 JP2018554450A JP2018554450A JP2019511240A5 JP 2019511240 A5 JP2019511240 A5 JP 2019511240A5 JP 2018554450 A JP2018554450 A JP 2018554450A JP 2018554450 A JP2018554450 A JP 2018554450A JP 2019511240 A5 JP2019511240 A5 JP 2019511240A5
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JP
Japan
Prior art keywords
neural stem
human neural
disease
stem cells
nucleotide sequence
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JP2018554450A
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English (en)
Japanese (ja)
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JP7197363B2 (ja
JP2019511240A (ja
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Priority claimed from PCT/US2017/027504 external-priority patent/WO2017180926A1/en
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Publication of JP2019511240A5 publication Critical patent/JP2019511240A5/ja
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JP2018554450A 2016-04-14 2017-04-13 ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集 Active JP7197363B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (3)

Publication Number Publication Date
JP2019511240A JP2019511240A (ja) 2019-04-25
JP2019511240A5 true JP2019511240A5 (OSRAM) 2020-05-14
JP7197363B2 JP7197363B2 (ja) 2022-12-27

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JP2018554450A Active JP7197363B2 (ja) 2016-04-14 2017-04-13 ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集

Country Status (8)

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

Families Citing this family (37)

* 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
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
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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors 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
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
CN110075319A (zh) * 2019-04-29 2019-08-02 白涛 一种抗遗传病生物导弹
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
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
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
CA3180217A1 (en) * 2020-06-02 2021-12-09 Gregory T. Bleck Nucleic acid constructs for protein manufacture
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
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
WO2023102712A1 (zh) * 2021-12-07 2023-06-15 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

Family Cites Families (11)

* 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
WO2004020597A2 (en) 2002-08-27 2004-03-11 Stemcells, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
WO2013158309A2 (en) 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
EP3138911B1 (en) * 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
ES2536353T3 (es) 2012-12-12 2015-05-22 The Broad Institute, Inc. Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
EP3057432B1 (en) 2013-10-17 2018-11-21 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
US9951353B2 (en) * 2013-11-15 2018-04-24 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Engineering neural stem cells using homologous recombination
EP3152319A4 (en) * 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design

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