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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- Prior art keywords
- neural stem
- human neural
- disease
- stem cells
- nucleotide sequence
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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 (cg-RX-API-DMAC7.html) | 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 |
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| US (2) | US20170298348A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3443085B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP7197363B2 (cg-RX-API-DMAC7.html) |
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| AU (2) | AU2017250683A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3020181A1 (cg-RX-API-DMAC7.html) |
| SG (1) | SG11201808920RA (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017180926A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (37)
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| CA2853829C (en) | 2011-07-22 | 2023-09-26 | 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| 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 |
| 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 |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| 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 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| 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 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | 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 |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | 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 |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst 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 |
| 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 |
| WO2021163327A1 (en) * | 2020-02-11 | 2021-08-19 | University Of Virginia Patent Foundation | Compositions and methods for treating and/or preventing ocular disorders |
| KR20220153021A (ko) * | 2020-03-11 | 2022-11-17 | 리모터 테라퓨틱스, 인코포레이티드 | 중추신경계를 통한 단백질을 확산시키기 위한 방법 및 재료 |
| 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 |
| US20230212590A1 (en) * | 2020-06-02 | 2023-07-06 | Catalent Pharma Solutions, Llc | 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)
| 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 |
| US7105150B2 (en) | 1999-02-12 | 2006-09-12 | Stemcells California, Inc. | In vivo screening methods using enriched neural stem cells |
| 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 |
| 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 |
| WO2013158309A2 (en) * | 2012-04-18 | 2013-10-24 | The Board Of Trustees Of The Leland Stanford Junior University | Non-disruptive gene targeting |
| GB2537000C (en) * | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| CA2891347C (en) * | 2012-12-06 | 2018-02-27 | Sigma-Aldrich Co. Llc | Crispr-based genome modification and regulation |
| PL2771468T3 (pl) * | 2012-12-12 | 2015-07-31 | Broad Inst Inc | Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami |
| WO2015057976A1 (en) * | 2013-10-17 | 2015-04-23 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
| 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 |
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2017
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- 2017-04-13 CN CN201780036763.3A patent/CN109312308A/zh active Pending
- 2017-04-13 WO PCT/US2017/027504 patent/WO2017180926A1/en not_active Ceased
- 2017-04-13 EP EP17783170.8A patent/EP3443085B1/en active Active
- 2017-04-13 AU AU2017250683A patent/AU2017250683A1/en not_active Abandoned
- 2017-04-13 CA CA3020181A patent/CA3020181A1/en active Pending
- 2017-04-13 SG SG11201808920RA patent/SG11201808920RA/en unknown
- 2017-04-13 JP JP2018554450A patent/JP7197363B2/ja active Active
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2023
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- 2023-07-24 AU AU2023208065A patent/AU2023208065A1/en not_active Abandoned
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