JP2018525979A5 - - Google Patents

Download PDF

Info

Publication number
JP2018525979A5
JP2018525979A5 JP2018501179A JP2018501179A JP2018525979A5 JP 2018525979 A5 JP2018525979 A5 JP 2018525979A5 JP 2018501179 A JP2018501179 A JP 2018501179A JP 2018501179 A JP2018501179 A JP 2018501179A JP 2018525979 A5 JP2018525979 A5 JP 2018525979A5
Authority
JP
Japan
Prior art keywords
cell
composition
nuclease
donor molecule
introducing
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.)
Granted
Application number
JP2018501179A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018525979A (ja
JP6954890B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/042024 external-priority patent/WO2017011519A1/en
Publication of JP2018525979A publication Critical patent/JP2018525979A/ja
Publication of JP2018525979A5 publication Critical patent/JP2018525979A5/ja
Priority to JP2020209010A priority Critical patent/JP7348160B2/ja
Application granted granted Critical
Publication of JP6954890B2 publication Critical patent/JP6954890B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018501179A 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 Active JP6954890B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020209010A JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562191918P 2015-07-13 2015-07-13
US62/191,918 2015-07-13
US201562247469P 2015-10-28 2015-10-28
US62/247,469 2015-10-28
US201662315438P 2016-03-30 2016-03-30
US62/315,438 2016-03-30
PCT/US2016/042024 WO2017011519A1 (en) 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020209010A Division JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Publications (3)

Publication Number Publication Date
JP2018525979A JP2018525979A (ja) 2018-09-13
JP2018525979A5 true JP2018525979A5 (enExample) 2019-08-22
JP6954890B2 JP6954890B2 (ja) 2021-10-27

Family

ID=57758305

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018501179A Active JP6954890B2 (ja) 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Country Status (7)

Country Link
US (2) US10450585B2 (enExample)
EP (1) EP3322297B1 (enExample)
JP (2) JP6954890B2 (enExample)
CN (1) CN108024544B (enExample)
AU (1) AU2016291778B2 (enExample)
CA (1) CA2991301A1 (enExample)
WO (1) WO2017011519A1 (enExample)

Families Citing this family (113)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
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
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
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
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
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
WO2017023801A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
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
PH12018500927B1 (en) 2015-10-28 2024-07-03 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
EP3443075B1 (en) 2016-04-15 2022-09-28 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
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
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
IL264639B2 (en) 2016-08-24 2024-01-01 Sangamo Therapeutics Inc Regulation of globulin gene expression using engineered zinc finger nucleases
KR20220145913A (ko) * 2016-08-24 2022-10-31 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CA3037086A1 (en) 2016-10-03 2018-04-12 Juno Therapeutics, Inc. Hpv-specific binding molecules
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
JP2020513248A (ja) 2016-10-19 2020-05-14 フロデザイン ソニックス, インク.Flodesign Sonics, Inc. 音響による親和性細胞抽出
EP3559214A1 (en) * 2016-12-21 2019-10-30 UCL Business PLC Therapeutic 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
BR112019015355A2 (pt) * 2017-01-26 2020-05-19 Sangamo Therapeutics Inc modificação genética de células b
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
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
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
AU2018283405B2 (en) 2017-06-15 2025-06-26 The Regents Of The University Of California Targeted non-viral DNA insertions
KR102708624B1 (ko) * 2017-06-16 2024-09-20 상가모 테라퓨틱스, 인코포레이티드 T 세포 및/또는 hla 수용체의 표적화된 파괴
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
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019006330A1 (en) * 2017-06-30 2019-01-03 Indiana University Research And Technology Corporation COMPOSITIONS AND METHODS FOR DETECTING REACTIVITY TO ALS
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
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
CN111194350A (zh) * 2017-09-18 2020-05-22 博雅辑因(北京)生物科技有限公司 一种基因编辑t细胞及其用途
AU2018335752B2 (en) * 2017-09-22 2025-04-10 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii
AU2018345539A1 (en) 2017-10-03 2020-04-16 Editas Medicine, Inc. HPV-specific binding molecules
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
EP3700924A4 (en) 2017-10-27 2021-11-03 The Regents of the University of California TARGETED REPLACEMENT OF ENDOGENOUS T-CELL RECEPTORS
JP2021505187A (ja) * 2017-12-11 2021-02-18 エディタス・メディシン、インコーポレイテッド 遺伝子編集のためのcpf1関連方法及び組成物
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
CN112585276A (zh) 2018-04-05 2021-03-30 朱诺治疗学股份有限公司 产生表达重组受体的细胞的方法和相关组合物
CA3095027A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. T cell receptors and engineered cells expressing same
JP7630280B2 (ja) 2018-04-05 2025-02-17 ジュノー セラピューティクス インコーポレイテッド 組換え受容体を発現するt細胞、関連ポリヌクレオチド、および方法
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
SG11202012044QA (en) 2018-06-29 2021-01-28 Wuhan Neurophth Biological Tech Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CA3105953A1 (en) 2018-07-09 2020-01-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
CN112805375A (zh) * 2018-08-07 2021-05-14 桑格摩生物治疗股份有限公司 用于治疗ii型黏多糖贮积症的方法
CN108949763A (zh) * 2018-08-15 2018-12-07 吉林大学 能有效抑制猪瘟病毒感染的精确突变LamR基因及应用
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020038352A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
SG11202103734PA (en) 2018-10-18 2021-05-28 Intellia Therapeutics Inc Compositions and methods for expressing factor ix
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
JP2022507453A (ja) * 2018-11-14 2022-01-18 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド T細胞送達のためのフソソーム組成物
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US11857641B2 (en) 2019-02-06 2024-01-02 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020223535A1 (en) 2019-05-01 2020-11-05 Juno Therapeutics, Inc. Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
BR112021021200A2 (pt) 2019-05-01 2021-12-21 Juno Therapeutics Inc Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos
BR112021022722A2 (pt) 2019-06-07 2022-01-04 Regeneron Pharma Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente
CN114502587A (zh) * 2019-07-30 2022-05-13 普瑞文森生物有限公司 通过非耗竭性b细胞抑制剂降低免疫原性的方法和组合物
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
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
US20230181641A1 (en) 2020-05-13 2023-06-15 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
CN116234558A (zh) 2020-06-26 2023-06-06 朱诺治疗学有限公司 条件性地表达重组受体的工程化t细胞、相关多核苷酸和方法
KR20230090367A (ko) 2020-11-04 2023-06-21 주노 쎄러퓨티크스 인코퍼레이티드 변형된 불변 cd3 이뮤노글로불린 슈퍼패밀리 쇄 유전자좌로부터 키메라 수용체를 발현하는 세포 및 관련 폴리뉴클레오티드 및 방법
CR20240195A (es) 2021-10-14 2024-06-20 Arsenal Biosciences Inc Células inmunitarias que tienen arnhc coexpresados y sistemas de compuerta lógica
WO2023081900A1 (en) 2021-11-08 2023-05-11 Juno Therapeutics, Inc. Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods
WO2023122038A1 (en) * 2021-12-20 2023-06-29 Washington University Methods and compositions for improved genetic therapies of multigenic diseases
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
WO2024161021A1 (en) 2023-02-03 2024-08-08 Juno Therapeutics Gmbh Methods for non-viral manufacturing of engineered immune cells
WO2024229281A1 (en) * 2023-05-02 2024-11-07 City Of Hope Adeno-associated virus compositions and methods of use thereof
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117858A1 (en) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplementation of liver enzyme expression
WO2025212519A1 (en) 2024-04-01 2025-10-09 Moonlight Bio, Inc. Dll3 binding proteins and uses thereof
US20250345431A1 (en) 2024-05-10 2025-11-13 Juno Therapeutics, Inc. Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US255250A (en) 1882-03-21 Whistling toy
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69535853D1 (de) 1994-03-23 2008-11-20 Univ Case Western Reserve Kompaktnukleinsäure und ihre verabreichung in zellen
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140815A (en) 1998-06-17 2000-10-31 Dover Instrument Corporation High stability spin stand platform
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
EP1129110B1 (en) * 1998-11-10 2009-12-09 Ludwig Institute For Cancer Research Platelet-derived growth factor d, dna coding therefor, and uses thereof
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
US6978476B2 (en) 2000-05-16 2005-12-20 Comsonics Device and method of determining location of signal ingress
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20030003583A1 (en) 2001-06-08 2003-01-02 Raphael Hirsch Regulation of transgene expression following AAV transduction
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
AU2003218382B2 (en) 2002-03-21 2007-12-13 Sangamo Therapeutics, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
GB0312086D0 (en) * 2003-05-27 2003-07-02 Novartis Ag Organic compounds
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
CA2561714A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
MXPA06011805A (es) * 2004-04-16 2006-12-15 Genentech Inc Metodo para aumentar agotamiento de celulas b.
EP2292274A1 (en) 2004-09-16 2011-03-09 Sangamo BioSciences, Inc. Compositions and methods for protein production
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
US8021867B2 (en) 2005-10-18 2011-09-20 Duke University Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝子不活性化のための方法と組成物
WO2007139898A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Variant foki cleavage half-domains
CN101679977B (zh) 2007-04-26 2013-05-01 桑格摩生物科学股份有限公司 向ppp1r12c基因座的靶向整合
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
JP2011518555A (ja) 2008-04-14 2011-06-30 サンガモ バイオサイエンシーズ, インコーポレイテッド 標的組込みのための線形ドナーコンストラクト
EP2313515B1 (en) 2008-08-22 2015-03-04 Sangamo BioSciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
KR101297192B1 (ko) 2008-11-10 2013-08-19 삼성전자주식회사 화상형성장치, 칩, 및, 칩 패키지
ES2498340T3 (es) 2008-11-17 2014-09-24 Koninklijke Nederlandse Akademie Van Wetenschappen Métodos para identificar compuestos de modulación de la linfangiogénesis, medios para ello, compuestos y usos de estos
AU2009322964B2 (en) 2008-12-04 2014-10-09 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
AU2009333863B2 (en) 2008-12-17 2015-02-05 Corteva Agriscience Llc Targeted integration into the Zp15 locus
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CA2788560A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
CA2788850C (en) 2010-02-09 2019-06-25 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
CN102821781A (zh) 2010-04-02 2012-12-12 赛拉基因公司 治疗帕金森氏病和脑多巴胺能神经元的其它病症的方法
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
US8772453B2 (en) 2010-05-03 2014-07-08 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
EP2596011B1 (en) 2010-07-21 2018-10-03 Sangamo Therapeutics, Inc. Methods and compositions for modification of a hla locus
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス, インコーポレイテッド Hprt遺伝子座を修飾するための方法および組成物
CA2854819C (en) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
CN102633883B (zh) * 2012-02-24 2014-06-25 上海白泽生物科技有限公司 一种能与egfr、her2、vegf高效结合的融合蛋白、其编码序列及用途
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
WO2013176915A1 (en) * 2012-05-25 2013-11-28 Roman Galetto Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014059173A2 (en) 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
WO2014089541A2 (en) 2012-12-07 2014-06-12 Haplomics, Inc. Factor viii mutation repair and tolerance induction
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US9999206B2 (en) 2013-07-25 2018-06-19 Utah State University Model and method for a transgenic bovidae expressing cardiac fibrosis and associated pathology
AU2014364051B2 (en) 2013-12-09 2018-12-20 Sangamo Therapeutics, Inc. Methods and compositions for treating hemophilia
WO2015127439A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
EP3909602A1 (en) 2014-04-25 2021-11-17 University of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
EP3139966B1 (en) 2014-05-08 2020-11-18 Sangamo Therapeutics, Inc. Compositions for use in treating huntington's disease
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
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
PH12018500927B1 (en) 2015-10-28 2024-07-03 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof

Similar Documents

Publication Publication Date Title
JP2018525979A5 (enExample)
JP7348160B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
Mangeot et al. Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins
JP6818720B2 (ja) カスパーゼポリペプチドを使用して部分的なアポトーシスを誘導するための方法
Yin et al. Delivery technologies for genome editing
US11186825B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting POU2AF1
US10280402B2 (en) Immune-compatible cells created by nuclease-mediated editing of genes encoding HLA
US11180730B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting GATA3
JP6722176B2 (ja) 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物
JP2024001104A (ja) 改変された細胞および治療の方法
US20190100801A1 (en) Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
JP2022531231A (ja) Cd8改変t細胞療法を使用するがんの治療のための組成物及び方法
JP7428712B2 (ja) 低/最小操作による遺伝子改変細胞の製造
JP2018171075A (ja) 改変されたカスパーゼポリペプチドおよびその使用
Rosigkeit et al. Monitoring translation activity of mRNA-loaded nanoparticles in mice
WO2017004153A1 (en) Tumor and microenvironment gene expression, compositions of matter and methods of use thereof
JP2016539627A5 (enExample)
CN114729383A (zh) 重组ad35载体及相关基因疗法改进
Meissner et al. Immune editing: overcoming immune barriers in stem cell transplantation
US20230000912A1 (en) Genetic, developmental and micro-environmental programs in idh-mutant gliomas, compositions of matter and methods of use thereof
KR102741572B1 (ko) 조직에서 코딩 리보핵산의 표적화된 전달, 발현 및 조절을 위한 조성물 및 방법
WO2022256448A2 (en) Compositions and methods for targeting, editing, or modifying genes
Zirger et al. Immune-mediated loss of transgene expression from virally transduced brain cells is irreversible, mediated by IFNγ, perforin, and TNFα, and due to the elimination of transduced cells
Zheng et al. Engineering of human mesenchymal stem cells resistant to multiple natural killer subtypes
JP2025518552A (ja) 細胞を操作するための組成物及び方法