JP2018518182A5 - - Google Patents

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Publication number
JP2018518182A5
JP2018518182A5 JP2017565277A JP2017565277A JP2018518182A5 JP 2018518182 A5 JP2018518182 A5 JP 2018518182A5 JP 2017565277 A JP2017565277 A JP 2017565277A JP 2017565277 A JP2017565277 A JP 2017565277A JP 2018518182 A5 JP2018518182 A5 JP 2018518182A5
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JP
Japan
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composition
cell
complex
cells
target dna
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JP2017565277A
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English (en)
Japanese (ja)
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JP2018518182A (ja
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Priority claimed from PCT/US2016/038161 external-priority patent/WO2016205703A1/en
Publication of JP2018518182A publication Critical patent/JP2018518182A/ja
Publication of JP2018518182A5 publication Critical patent/JP2018518182A5/ja
Pending legal-status Critical Current

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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

Publications (2)

Publication Number Publication Date
JP2018518182A JP2018518182A (ja) 2018-07-12
JP2018518182A5 true JP2018518182A5 (enExample) 2019-07-18

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ID=57546388

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Application Number Title Priority Date Filing Date
JP2017565277A Pending JP2018518182A (ja) 2015-06-17 2016-06-17 ゲノム編集のためのcrispr/cas9複合体

Country Status (8)

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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)
ZA (1) ZA201800026B (enExample)

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

Family Cites Families (22)

* 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
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
BR112017020750A2 (en) * 2015-03-27 2018-06-26 President And Fellows Of Harvard College modified t-cells and methods of producing and using them
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

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