JP2018520648A - 初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上 - Google Patents

初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上 Download PDF

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JP2018520648A
JP2018520648A JP2017559075A JP2017559075A JP2018520648A JP 2018520648 A JP2018520648 A JP 2018520648A JP 2017559075 A JP2017559075 A JP 2017559075A JP 2017559075 A JP2017559075 A JP 2017559075A JP 2018520648 A JP2018520648 A JP 2018520648A
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nucleic acid
cell
acid sequence
protein
cells
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JP2018520648A5 (cg-RX-API-DMAC7.html
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シャーレンバーグ，アンドリュー
ローリングス，デイヴィッド
ジェンセン，マイケル，シー．
グヴィャズダ，カミラ
グリアー，アレクサンドラ
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シアトル チルドレンズ ホスピタル， ディービーエー シアトル チルドレンズ リサーチ インスティテュート
シアトル チルドレンズ ホスピタル， ディービーエー シアトル チルドレンズ リサーチ インスティテュート
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JP2017559075A 2015-05-13 2016-05-12 初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上 Ceased JP2018520648A (ja)

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US201562161104P 2015-05-13 2015-05-13
US62/161,104 2015-05-13
PCT/US2016/032153 WO2016183345A1 (en) 2015-05-13 2016-05-12 Enhancing endonuclease based gene editing in primary cells

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JP2018520648A true JP2018520648A (ja) 2018-08-02
JP2018520648A5 JP2018520648A5 (cg-RX-API-DMAC7.html) 2019-06-13

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US (3) US10563226B2 (cg-RX-API-DMAC7.html)
EP (1) EP3294343A4 (cg-RX-API-DMAC7.html)
JP (1) JP2018520648A (cg-RX-API-DMAC7.html)
CN (1) CN108136047B (cg-RX-API-DMAC7.html)
AU (1) AU2016262093B2 (cg-RX-API-DMAC7.html)
CA (1) CA2985650A1 (cg-RX-API-DMAC7.html)
HK (1) HK1252144A1 (cg-RX-API-DMAC7.html)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024503249A (ja) * 2020-12-21 2024-01-25 ２セブンティ バイオ インコーポレイテッド 部位特異的変異導入のための組成物及び方法

Families Citing this family (75)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
EP3092049A1 (en) 2014-01-08 2016-11-16 Flodesign Sonics Inc. Acoustophoresis device with dual acoustophoretic chamber
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US10166255B2 (en) 2015-07-31 2019-01-01 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US11459586B2 (en) * 2016-04-19 2022-10-04 President And Fellows Of Harvard College Methods for increasing efficiency of nuclease-mediated gene editing in stem cells
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (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
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
GB2604416B (en) 2016-10-18 2023-03-15 Univ Minnesota Tumor infiltating lymphocytes and methods of therapy
CN110494543A (zh) 2016-10-19 2019-11-22 弗洛设计声能学公司 通过声学的亲和细胞提取
AU2017347848A1 (en) * 2016-10-27 2019-05-23 Intima Bioscience, Inc. Viral methods of making genetically modified cells
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
CN107746845B (zh) * 2016-12-28 2021-03-26 北京微旋基因技术有限公司 特异性靶向LAG-3基因的sgRNA和特异性敲除LAG-3基因的方法
CN107760680B (zh) * 2016-12-28 2020-09-22 北京微旋基因技术有限公司 特异性靶向TIM-3基因的sgRNA和特异性敲除TIM-3基因的方法
WO2018165504A1 (en) 2017-03-09 2018-09-13 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
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
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
CN108866100A (zh) * 2017-05-16 2018-11-23 中国科学院上海生命科学研究院 一种高效率的基因编辑方法
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
EP3645036A1 (en) 2017-06-30 2020-05-06 Cellectis Cellular immunotherapy for repetitive administration
US11732274B2 (en) 2017-07-28 2023-08-22 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
EP3701028B8 (en) 2017-10-24 2024-10-23 Editas Medicine, Inc. Systems and methods for treating hyper-igm syndrome
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． 音響トランスデューサドライバ及びコントローラ
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20190284553A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
WO2019189573A1 (ja) * 2018-03-29 2019-10-03 学校法人自治医科大学 ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法
AU2019261385A1 (en) * 2018-04-27 2020-11-19 Seattle Children's Hospital (dba Seattle Children's Research Institute) Therapeutic genome editing in X-linked hyper IgM syndrome
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US20210299174A1 (en) * 2018-05-30 2021-09-30 M2X2 Therapeutics, Inc. Cell therapy
CN112601812A (zh) * 2018-06-25 2021-04-02 圣拉斐尔医院有限责任公司 基因疗法
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN109528653B (zh) * 2018-11-22 2020-07-31 北京大学 具有基因编辑功能的膜性囊泡及其制备方法、药物组合物和用途
CN109735516B (zh) * 2019-01-22 2021-09-03 自然资源部第一海洋研究所 受核苷酸片段引导具有特异核酸内切酶活性的piwi蛋白
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
CN111812066B (zh) * 2019-04-10 2023-04-28 华东理工大学 基于CRISPR/Cas12a系统的生物传感器、试剂盒及其在小分子检测中的用途
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111909961B (zh) * 2020-08-21 2022-04-19 华侨大学 一种CRISPR/Cas介导的ATL细胞基因编辑载体及其应用
WO2022170193A2 (en) * 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy
JP2024505678A (ja) * 2021-02-08 2024-02-07 インテリア セラピューティクス，インコーポレイテッド 免疫療法のためのリンパ球活性化遺伝子３（ｌａｇ３）組成物及び方法
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
EP4392060A1 (en) * 2021-08-24 2024-07-03 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
US20250122535A1 (en) * 2021-12-03 2025-04-17 The General Hospital Corporation Crispr-associated transposases and methods of use thereof
WO2023122036A1 (en) * 2021-12-20 2023-06-29 Sobol Robert E Methods and compositions for improved molecular therapies of multigenic diseases
EP4590818A2 (en) * 2022-09-19 2025-07-30 Emendobio Inc. Biallelic knockout of pdcd1
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
WO2024107119A1 (en) * 2022-11-17 2024-05-23 Agency For Science, Technology And Research T3 vectors for recombinant protein production in mammalian cells
CN117987436B (zh) * 2024-04-03 2024-06-25 南京鸿明生物科技有限公司 一种双链靶dna序列的制备方法
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004505633A (ja) * 2000-08-03 2004-02-26 オニックス　ファーマシューティカルズ，インコーポレイティド アデノウイルスｅ１ｂ−５５ｋ単一アミノ酸変異体および使用方法
WO2008153421A1 (en) * 2007-06-15 2008-12-18 Otago Innovation Limited Anticancer therapies
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
US8999641B2 (en) * 2012-12-12 2015-04-07 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2016057850A1 (en) * 2014-10-09 2016-04-14 Seattle Children' S Hospital (Dba Seattle Children' S Research Institute) Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7838219B2 (en) 2001-02-02 2010-11-23 Novici Biotech Llc Method of increasing complementarity in a heteroduplex
CA2514517A1 (en) 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
EP2412806B1 (en) 2010-07-28 2014-01-08 Institut Pasteur Use of terminal deoxynucleotidyl transferase for mutagenic DNA repair to generate variability, at a determined position in DNA
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
US9044492B2 (en) 2011-02-04 2015-06-02 Cellectis Sa Method for modulating the efficiency of double-strand break-induced mutagenesis
US9540623B2 (en) 2011-07-08 2017-01-10 Cellectis Method for increasing the efficiency of double-strand-break induced mutagenesis
WO2013033627A2 (en) * 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
KR20160060659A (ko) * 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
US20160237455A1 (en) * 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
AU2014337248B2 (en) * 2013-10-17 2020-09-24 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3256170B1 (en) * 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004505633A (ja) * 2000-08-03 2004-02-26 オニックス　ファーマシューティカルズ，インコーポレイティド アデノウイルスｅ１ｂ−５５ｋ単一アミノ酸変異体および使用方法
WO2008153421A1 (en) * 2007-06-15 2008-12-18 Otago Innovation Limited Anticancer therapies
US8999641B2 (en) * 2012-12-12 2015-04-07 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
WO2016057850A1 (en) * 2014-10-09 2016-04-14 Seattle Children' S Hospital (Dba Seattle Children' S Research Institute) Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BAKER, A., ET AL.: ""Adenovirus E4 34k and E1b 55k oncoproteins target host DNA ligase IV for proteasomal degradation."", JOURNAL OF VIROLOGY, vol. 81, no. 13, JPN6020020080, July 2007 (2007-07-01), pages 7034 - 7040, ISSN: 0004656026 *
CHU, V.T., ET AL.: ""Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing", NATURE BIOTECHNOLOGY, vol. 33, no. 5, JPN6020020079, May 2015 (2015-05-01), pages 543 - 548, ISSN: 0004514784 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024503249A (ja) * 2020-12-21 2024-01-25 ２セブンティ バイオ インコーポレイテッド 部位特異的変異導入のための組成物及び方法

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