CN107949641A - 用于基因组编辑的crispr/cas9复合物 - Google Patents

用于基因组编辑的crispr/cas9复合物 Download PDF

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CN107949641A
CN107949641A CN201680048445.4A CN201680048445A CN107949641A CN 107949641 A CN107949641 A CN 107949641A CN 201680048445 A CN201680048445 A CN 201680048445A CN 107949641 A CN107949641 A CN 107949641A
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cells
cell
lys
complex
leu
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蒂姆·汤斯
丁磊
詹家伟
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UAB Research Foundation
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CN201680048445.4A 2015-06-17 2016-06-17 用于基因组编辑的crispr/cas9复合物 Pending CN107949641A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562181138P 2015-06-17 2015-06-17
US62/181,138 2015-06-17
US201562266316P 2015-12-11 2015-12-11
US62/266,316 2015-12-11
PCT/US2016/038161 WO2016205703A1 (en) 2015-06-17 2016-06-17 Crispr/cas9 complex for genomic editing

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US (2) US11643668B2 (enExample)
EP (1) EP3310932B1 (enExample)
JP (1) JP2018518182A (enExample)
CN (1) CN107949641A (enExample)
AU (1) AU2016278982A1 (enExample)
CA (1) CA2989831A1 (enExample)
WO (1) WO2016205703A1 (enExample)
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Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110283838A (zh) * 2019-06-27 2019-09-27 安徽省农业科学院水稻研究所 一种高剪切效率ScCas9基因及其应用
WO2019227640A1 (zh) * 2018-06-01 2019-12-05 上海科技大学 利用碱基编辑修复fbn1t7498c突变的试剂和方法
WO2021037234A1 (zh) * 2019-08-29 2021-03-04 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
CN112442516A (zh) * 2019-08-29 2021-03-05 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
CN113039278A (zh) * 2018-10-31 2021-06-25 诺维信公司 通过指导的内切核酸酶和单链寡核苷酸进行基因组编辑
CN116497003A (zh) * 2023-06-29 2023-07-28 北京百普赛斯生物科技股份有限公司 一种从溶液中纯化目标蛋白的方法

Families Citing this family (44)

* 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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2017053729A1 (en) * 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of 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
EP3497214B1 (en) 2016-08-09 2023-06-28 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
EP3526320A1 (en) 2016-10-14 2019-08-21 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
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018182361A1 (ko) * 2017-03-31 2018-10-04 한국과학기술원 CRISPR/Cas 시스템과 재조합 효소 및 단일가닥 올리고디옥시리보핵산을 이용한 코리네박테리움 변이균주 제조방법
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
EP3638317A4 (en) 2017-06-15 2021-03-17 The Regents of The University of California TARGETED NONVIRAL DNA INSERTIONS
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
KR102770787B1 (ko) 2017-10-27 2025-02-19 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
WO2019118949A1 (en) 2017-12-15 2019-06-20 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
US20210071188A1 (en) * 2018-05-29 2021-03-11 Monsanto Technology Llc Transgenic plants with enhanced traits
US12281338B2 (en) 2018-10-29 2025-04-22 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.
RU2707542C1 (ru) 2019-03-28 2019-11-27 Федеральное бюджетное учреждение науки "Центральный научно-исследовательский институт эпидемиологии" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ЦНИИ Эпидемиологии Роспотребнадзора) Способ получения препарата рекомбинантной нуклеазы CAS, по существу, свободного от бактериальных эндотоксинов, полученный данным способом препарат и содержащий его набор для использования в системе CRISPR/Cas
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020219775A1 (en) * 2019-04-24 2020-10-29 Magenta Therapeutics, Inc. Anti-cd117 antibody-drug conjugates and uses thereof
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
KR102421847B1 (ko) * 2020-11-03 2022-07-18 한국생명공학연구원 Jak3 유전자 돌연변이 중증복합면역결핍 동물모델 및 이의 제조방법
PE20241173A1 (es) 2021-10-14 2024-05-28 Arsenal Biosciences Inc Celulas inmunitarias que tienen arnhc coexpresados y sistemas de compuerta logica
EP4551696A2 (en) * 2022-07-08 2025-05-14 The Regents of the University of California Modified crispr-cas effector polypeptides and methods of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071903A1 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Use of cationic lipids to deliver cas9

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012525146A (ja) 2009-04-28 2012-10-22 プレジデント アンド フェロウズ オブ ハーバード カレッジ 細胞透過のための過剰に荷電されたタンパク質
SG11201405103SA (en) * 2012-02-24 2014-09-26 Hutchinson Fred Cancer Res Compositions and methods for the treatment of hemoglobinopathies
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
ES2824024T3 (es) * 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
CA2894668A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN119752887A (zh) 2012-12-12 2025-04-04 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
CA3185368A1 (en) 2013-04-03 2014-10-09 Memorial Sloan-Kettering Cancer Center Effective generation of tumor-targeted t cells derived from pluripotent stem cells
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP3309248B1 (en) 2013-05-29 2021-06-09 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3250693B2 (en) 2015-01-30 2023-12-20 The Regents of The University of California Protein delivery in primary hematopoietic cells
BR112017020750A2 (pt) 2015-03-27 2018-06-26 Harvard College células t modificadas e métodos de produção e utilização das mesmas
CN104894068A (zh) 2015-05-04 2015-09-09 南京凯地生物科技有限公司 一种利用CRISPR/Cas9制备CAR-T细胞的方法
CA2989858A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
ES2933961T3 (es) 2016-04-15 2023-02-15 Memorial Sloan Kettering Cancer Center Células T transgénicas y composiciones de células T de receptor de antígeno quimérico y métodos relacionados

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071903A1 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Use of cationic lipids to deliver cas9

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
BIALK ET AL: "Regulation of Gene Editing Activity Directed by Single-Stranded Oligonucleotides and CRISPR/Cas9 Systems", 《PLOS ONE》 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019227640A1 (zh) * 2018-06-01 2019-12-05 上海科技大学 利用碱基编辑修复fbn1t7498c突变的试剂和方法
CN113039278A (zh) * 2018-10-31 2021-06-25 诺维信公司 通过指导的内切核酸酶和单链寡核苷酸进行基因组编辑
CN110283838A (zh) * 2019-06-27 2019-09-27 安徽省农业科学院水稻研究所 一种高剪切效率ScCas9基因及其应用
CN110283838B (zh) * 2019-06-27 2022-03-25 安徽省农业科学院水稻研究所 一种高剪切效率ScCas9基因及其应用
WO2021037234A1 (zh) * 2019-08-29 2021-03-04 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
CN112442516A (zh) * 2019-08-29 2021-03-05 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
CN114222816A (zh) * 2019-08-29 2022-03-22 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
CN116497003A (zh) * 2023-06-29 2023-07-28 北京百普赛斯生物科技股份有限公司 一种从溶液中纯化目标蛋白的方法

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