JP2018518182A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
composition
cell
complex
cells
target dna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2017565277A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018518182A (ja
Filing date
Publication date
Application filed filed Critical
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

Links

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 (https=) 2019-07-18

Family

ID=57546388

Family Applications (1)

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)

Country Link
US (2) US11643668B2 (https=)
EP (1) EP3310932B1 (https=)
JP (1) JP2018518182A (https=)
CN (1) CN107949641A (https=)
AU (1) AU2016278982A1 (https=)
CA (1) CA2989831A1 (https=)
WO (1) WO2016205703A1 (https=)
ZA (1) ZA201800026B (https=)

Families Citing this family (51)

* 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
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
AU2016261358B2 (en) 2015-05-11 2021-09-16 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
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したCas9蛋白質
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 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
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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
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
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising 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
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
CA3067382A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral 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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
KR102503130B1 (ko) 2017-10-27 2023-02-24 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
EP3797160A1 (en) 2018-05-23 2021-03-31 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
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
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
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
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
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3958909A4 (en) * 2019-04-24 2024-01-10 Magenta Therapeutics, Inc. ANTI-CD117 ANTIBODY DRUG CONJUGATES AND USES THEREOF
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
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
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
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
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
KR102421847B1 (ko) * 2020-11-03 2022-07-18 한국생명공학연구원 Jak3 유전자 돌연변이 중증복합면역결핍 동물모델 및 이의 제조방법
AU2022366987A1 (en) 2021-10-14 2024-05-16 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
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
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

Family Cites Families (22)

* 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
RU2650811C2 (ru) 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2906684B8 (en) 2012-10-10 2020-09-02 Sangamo Therapeutics, 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
CN105121648B (zh) 2012-12-12 2021-05-07 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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
AU2014248119B2 (en) 2013-04-03 2019-06-20 Memorial Sloan-Kettering Cancer Center Effective generation of tumor-targeted T-cells derived from pluripotent stem cells
JP2016522679A (ja) 2013-04-04 2016-08-04 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系を用いたゲノム編集の治療的使用
WO2014191128A1 (en) 2013-05-29 2014-12-04 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
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)
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
KR20260048600A (ko) * 2015-03-27 2026-04-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 변형된 t 세포 및 이의 제조 및 사용 방법
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
US11377637B2 (en) 2016-04-15 2022-07-05 Memorial Sloan Kettering Cancer Center Transgenic T cell and chimeric antigen receptor T cell compositions and related methods

Similar Documents

Publication Publication Date Title
JP2018518182A5 (https=)
JP7698828B2 (ja) Rnaを編集する方法および組成物
US20230203541A1 (en) Optimized gene editing utilizing a recombinant endonuclease system
JP2018518181A5 (https=)
JP2023078373A (ja) Rnaを編集する方法および組成物
US20220090127A1 (en) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
JP2016537028A5 (https=)
JP2017513477A5 (https=)
JP2019524149A5 (https=)
JP2018512162A5 (https=)
US20250084437A1 (en) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
WO2018117746A1 (ko) 동물 배아의 염기 교정용 조성물 및 염기 교정 방법
US20190169653A1 (en) Method for preparing gene knock-in cells
IL274161B1 (en) Systems and methods for treating hyper-IgM syndrome
KR20210116531A (ko) 영양 장애형 표피 수포증 치료약
US20200263206A1 (en) Targeted integration systems and methods for the treatment of hemoglobinopathies
WO2021030756A1 (en) Rna-guided dna integration and modification
CN109929865B (zh) 基于gal4-uas系统的crispr辅助反式增强子激活基因表达的方法及其应用
Wong Gene targeting and genome editing
JP2023507034A (ja) ゲノム編集効率を向上させる融合タンパク質及びその使用
WO2021195532A1 (en) Genome engineering using crispr rna-guided integrases
CA3253302A1 (en) CELL COMPOSITIONS AND ENGINEERING METHODS
EP4230737A1 (en) Novel enhanced base editing or revising fusion protein and use thereof
US20210071202A1 (en) Genome editing method, composition, cell, cell preparation, and method for producing cell preparation
IL300563A (en) Nuclease-mediated nucleic acid modification