JP6450371B2 - Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途 - Google Patents

Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途 Download PDF

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JP6450371B2
JP6450371B2 JP2016516173A JP2016516173A JP6450371B2 JP 6450371 B2 JP6450371 B2 JP 6450371B2 JP 2016516173 A JP2016516173 A JP 2016516173A JP 2016516173 A JP2016516173 A JP 2016516173A JP 6450371 B2 JP6450371 B2 JP 6450371B2
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アレクサンダー アストラカン
アレクサンダー アストラカン
ジョーダン ジャージャー
ジョーダン ジャージャー
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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JP2016516173A 2013-05-31 2014-05-28 Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途 Active JP6450371B2 (ja)

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DKPA201370303 2013-05-31
DKPA201370303 2013-05-31
PCT/EP2014/061189 WO2014191527A1 (en) 2013-05-31 2014-05-28 A laglidadg homing endonuclease cleaving the t cell receptor alpha gene and uses thereof

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Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012138927A2 (en) * 2011-04-05 2012-10-11 Philippe Duchateau Method for the generation of compact tale-nucleases and uses thereof
US10323236B2 (en) 2011-07-22 2019-06-18 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
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
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
EP4079847A1 (en) 2014-07-30 2022-10-26 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
AU2016333898B2 (en) * 2015-10-05 2020-11-12 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
ES2768984T3 (es) 2015-10-05 2020-06-24 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
BR112018011089A2 (pt) 2015-12-04 2018-12-04 Intellia Therapeutics Inc composições e métodos para a imuno-oncologia
BR112018068354A2 (pt) * 2016-03-11 2019-01-15 Bluebird Bio Inc células efetoras imunológicas do genoma editado
CR20180503A (es) * 2016-04-14 2018-12-21 Hutchinson Fred Cancer Res Composiciones y métodos para programar células terapéuticas utilizando nanoportadores de ácidos nucleicos dirigidos
US10188749B2 (en) 2016-04-14 2019-01-29 Fred Hutchinson Cancer Research Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3487994A4 (en) * 2016-07-25 2020-01-29 Bluebird Bio, Inc. HOMING BCL11A ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US20190309274A1 (en) 2016-08-16 2019-10-10 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018039333A1 (en) * 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
DK3510157T5 (da) * 2016-09-08 2024-09-02 2Seventy Bio Inc Pd-1-homing-endonucleasevarianter, sammensætninger og fremgangsmåder til anvendelse
MA46717A (fr) 2016-10-11 2019-09-11 Bluebird Bio Inc Variantes de l'endonucléase homing tcra
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
AU2017346683B2 (en) * 2016-10-17 2024-04-18 Regeneron Pharmaceuticals, Inc. TGFβR2 endonuclease variants, compositions, and methods of use
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
KR20250072712A (ko) 2016-12-02 2025-05-26 앤젤레스 테라퓨틱스, 인코포레이티드 합성 면역 수용체 및 이의 사용 방법
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US11566061B2 (en) 2017-01-05 2023-01-31 Fred Hutchinson Cancer Center Systems and methods to improve vaccine efficacy
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
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
JP2020513832A (ja) 2017-03-22 2020-05-21 ノバルティス アーゲー 免疫腫瘍学のための組成物および方法
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
WO2018189360A1 (en) 2017-04-13 2018-10-18 Cellectis New sequence specific reagents targeting ccr5 in primary hematopoietic cells
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
KR102590466B1 (ko) * 2017-05-25 2023-10-19 2세븐티 바이오, 인코포레이티드 Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
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
WO2019070974A1 (en) * 2017-10-04 2019-04-11 Bluebird Bio, Inc. PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
CN111479830A (zh) * 2017-10-10 2020-07-31 国立大学法人广岛大学 使用铂talen的t细胞受体的完全取代技术
CN111448312A (zh) 2017-10-10 2020-07-24 国立大学法人广岛大学 使用效应T细胞(Teff)抗原受体的抗原特异性调节性T细胞(Treg)的制作技术
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US20210002621A1 (en) * 2017-12-20 2021-01-07 Bluebird Bio, Inc. Ctla4 homing endonuclease variants, compositions, and methods of use
KR102617818B1 (ko) 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
BR112020025439A2 (pt) 2018-06-14 2021-03-16 Bluebird Bio, Inc. Receptores de antígenos quiméricos cd79a
WO2020072059A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CA3122278A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
AU2020253538A1 (en) 2019-04-03 2021-11-18 Precision Biosciences, Inc. Genetically-modified immune cells comprising a microRNA-adapted shRNA (shRNAmiR)
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021016608A1 (en) 2019-07-25 2021-01-28 Precision Biosciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
JP2022544825A (ja) 2019-08-20 2022-10-21 プレシジョン バイオサイエンシズ，インク． 細胞免疫療法のためのリンパ球枯渇投与レジメン
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
WO2021087305A1 (en) 2019-10-30 2021-05-06 Precision Biosciences, Inc. Cd20 chimeric antigen receptors and methods of use for immunotherapy
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP4192875A1 (en) 2020-08-10 2023-06-14 Precision BioSciences, Inc. Antibodies and fragments specific for b-cell maturation antigen and uses thereof
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
EP4284823A1 (en) 2021-01-28 2023-12-06 Precision BioSciences, Inc. Modulation of tgf beta signaling in genetically-modified eukaryotic cells
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023081767A1 (en) 2021-11-05 2023-05-11 Precision Biosciences, Inc. Methods for immunotherapy
IL312721A (en) 2021-11-23 2024-07-01 Cellectis Sa New tale protein scaffolds with improved on-target/off-target activity ratios
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
AU2024206812A1 (en) 2023-01-05 2025-08-14 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8956828B2 (en) * 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
US20140148361A1 (en) * 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
WO2012138927A2 (en) * 2011-04-05 2012-10-11 Philippe Duchateau Method for the generation of compact tale-nucleases and uses thereof
EP2729567B1 (en) * 2011-07-08 2016-10-05 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenssis
WO2013068845A2 (en) * 2011-11-07 2013-05-16 The University Of Western Ontario Endonuclease for genome editing

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EP3004338A1 (en) 2016-04-13
WO2014191527A1 (en) 2014-12-04
CA2913872A1 (en) 2014-12-04
AU2014273091A1 (en) 2015-12-03
US20160130569A1 (en) 2016-05-12
HK1223394A1 (en) 2017-07-28
EP3004338B1 (en) 2019-01-02
US10000746B2 (en) 2018-06-19
CA2913872C (en) 2022-01-18
AU2014273091B2 (en) 2019-12-12
ES2716867T3 (es) 2019-06-17
JP2016520320A (ja) 2016-07-14
DK3004338T3 (en) 2019-04-08

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