JP2018518182A - ゲノム編集のためのｃｒｉｓｐｒ／ｃａｓ９複合体 - Google Patents

ゲノム編集のためのｃｒｉｓｐｒ／ｃａｓ９複合体 Download PDF

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JP2018518182A
JP2018518182A JP2017565277A JP2017565277A JP2018518182A JP 2018518182 A JP2018518182 A JP 2018518182A JP 2017565277 A JP2017565277 A JP 2017565277A JP 2017565277 A JP2017565277 A JP 2017565277A JP 2018518182 A JP2018518182 A JP 2018518182A
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cells
complex
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JP2018518182A5 (cg-RX-API-DMAC10.html
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ティム タウンズ，
ティム タウンズ，
レイ ディン，
レイ ディン，
チャ−ウェイ チャン，
チャ−ウェイ チャン，
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ザ ユーエービー リサーチ ファンデーション
ザ ユーエービー リサーチ ファンデーション
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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JP2017565277A 2015-06-17 2016-06-17 ゲノム編集のためのｃｒｉｓｐｒ／ｃａｓ９複合体 Pending JP2018518182A (ja)

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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JP2018518182A true JP2018518182A (ja) 2018-07-12
JP2018518182A5 JP2018518182A5 (cg-RX-API-DMAC10.html) 2019-07-18

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US (2) US11643668B2 (cg-RX-API-DMAC10.html)
EP (1) EP3310932B1 (cg-RX-API-DMAC10.html)
JP (1) JP2018518182A (cg-RX-API-DMAC10.html)
CN (1) CN107949641A (cg-RX-API-DMAC10.html)
AU (1) AU2016278982A1 (cg-RX-API-DMAC10.html)
CA (1) CA2989831A1 (cg-RX-API-DMAC10.html)
WO (1) WO2016205703A1 (cg-RX-API-DMAC10.html)
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Families Citing this family (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 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
US11911415B2 (en) 2015-06-09 2024-02-27 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
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 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
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
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
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
SG11202003798TA (en) 2017-10-27 2020-05-28 Univ California Targeted replacement of endogenous t cell receptors
US12406749B2 (en) 2017-12-15 2025-09-02 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
AR114534A1 (es) 2018-05-29 2020-09-16 Monsanto Technology Llc Plantas transgénicas con rasgos mejorados
CN108753778B (zh) * 2018-06-01 2021-11-02 上海科技大学 利用碱基编辑修复fbn1t7498c突变的试剂和方法
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3874051A1 (en) * 2018-10-31 2021-09-08 Novozymes A/S Genome editing by guided endonuclease and single-stranded oligonucleotide
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
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
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022530438A (ja) * 2019-04-24 2022-06-29 マジェンタ セラピューティクス インコーポレイテッド 抗ｃｄ１１７抗体薬物複合体およびその使用
US12667087B2 (en) * 2019-06-07 2026-06-30 Agios Pharmaceuticals, Inc. Methods of treatment with aminolevulinic acid synthase 2 (ALAS2) modulators
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
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
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
KR102421847B1 (ko) * 2020-11-03 2022-07-18 한국생명공학연구원 Jak3 유전자 돌연변이 중증복합면역결핍 동물모델 및 이의 제조방법
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados y sistemas de compuerta lógica.
WO2024011176A2 (en) * 2022-07-08 2024-01-11 The Regents Of The University Of California Modified crispr-cas effector polypeptides and methods of use thereof
CN116497003B (zh) * 2023-06-29 2026-07-14 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006498A2 (en) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2015035136A2 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2016160721A1 (en) * 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129023A2 (en) 2009-04-28 2010-11-11 President And Fellows Of Harvard College Supercharged proteins for cell penetration
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2014059173A2 (en) 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
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
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
EP3789487A1 (en) 2013-04-03 2021-03-10 Memorial Sloan Kettering Cancer Center Effective generation of tumor-targeted t-cells derived from pluripotent stem cells
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6608807B2 (ja) 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
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)
US20190388469A1 (en) 2015-01-30 2019-12-26 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
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
US11555207B2 (en) 2015-06-17 2023-01-17 The Uab Research Foundation CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
KR102614675B1 (ko) 2016-04-15 2023-12-19 메모리얼 슬로안 케터링 캔서 센터 유전자이식 t 세포 및 키메라 항원 수용체 t 세포 조성물 및 관련 방법

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006498A2 (en) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2015035136A2 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2016160721A1 (en) * 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BARRANGOU R. ET AL., MOLECULAR CELL, vol. 54 (2014), JPN6021008201, pages 234 - 244, ISSN: 0004619136 *
CRONICAN J. ET AL., ACS CHEMICAL BIOLOGY, vol. Vol.5 No.8 (2010), JPN6020025333, pages 747 - 752, ISSN: 0004619133 *
LIN S. ET AL., ELIFE, vol. 3(2014): e04766, JPN6020025337, ISSN: 0004619135 *
MANSILLA-SOTO J. ET AL.: "Repair of the sickle-cell disease mutation in human stem cells using CRISPR/Cas9 and single-stranded", MOLECULAR THERAPY, vol. Vol.23 Supplement 1 (2015 May), JPN6020025335, pages 137 - 343, ISSN: 0004460562 *
NOURI F. S. ET AL., BIOMACROMOLECULES, vol. 14 (2013), JPN6020025334, pages 2033 - 2040, ISSN: 0004619134 *

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