JP2017504354A5 - - Google Patents

Download PDF

Info

Publication number
JP2017504354A5
JP2017504354A5 JP2016557544A JP2016557544A JP2017504354A5 JP 2017504354 A5 JP2017504354 A5 JP 2017504354A5 JP 2016557544 A JP2016557544 A JP 2016557544A JP 2016557544 A JP2016557544 A JP 2016557544A JP 2017504354 A5 JP2017504354 A5 JP 2017504354A5
Authority
JP
Japan
Prior art keywords
zinc finger
pharmaceutical composition
vector
protein
aav vector
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
JP2016557544A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017504354A (ja
JP6642943B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2014/069307 external-priority patent/WO2015089046A1/en
Publication of JP2017504354A publication Critical patent/JP2017504354A/ja
Publication of JP2017504354A5 publication Critical patent/JP2017504354A5/ja
Application granted granted Critical
Publication of JP6642943B2 publication Critical patent/JP6642943B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2016557544A 2013-12-09 2014-12-09 血友病を処置するための方法および組成物 Expired - Fee Related JP6642943B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361913838P 2013-12-09 2013-12-09
US61/913,838 2013-12-09
US201461943884P 2014-02-24 2014-02-24
US61/943,884 2014-02-24
PCT/US2014/069307 WO2015089046A1 (en) 2013-12-09 2014-12-09 Methods and compositions for treating hemophilia

Publications (3)

Publication Number Publication Date
JP2017504354A JP2017504354A (ja) 2017-02-09
JP2017504354A5 true JP2017504354A5 (OSRAM) 2017-12-21
JP6642943B2 JP6642943B2 (ja) 2020-02-12

Family

ID=53270543

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2016557544A Expired - Fee Related JP6642943B2 (ja) 2013-12-09 2014-12-09 血友病を処置するための方法および組成物
JP2016557545A Expired - Fee Related JP6535684B2 (ja) 2013-12-09 2014-12-09 ゲノム操作のための方法および組成物
JP2018236131A Pending JP2019037254A (ja) 2013-12-09 2018-12-18 ゲノム操作のための方法および組成物

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2016557545A Expired - Fee Related JP6535684B2 (ja) 2013-12-09 2014-12-09 ゲノム操作のための方法および組成物
JP2018236131A Pending JP2019037254A (ja) 2013-12-09 2018-12-18 ゲノム操作のための方法および組成物

Country Status (11)

Country Link
US (6) US10081661B2 (OSRAM)
EP (3) EP3757116A1 (OSRAM)
JP (3) JP6642943B2 (OSRAM)
CN (3) CN111330021A (OSRAM)
AU (3) AU2014363991B2 (OSRAM)
CA (3) CA2931848A1 (OSRAM)
DK (1) DK3080274T3 (OSRAM)
ES (1) ES2813367T3 (OSRAM)
HU (1) HUE051628T2 (OSRAM)
IL (3) IL245785B (OSRAM)
WO (2) WO2015089046A1 (OSRAM)

Families Citing this family (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
CA2931848A1 (en) * 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3110454B1 (en) 2014-02-24 2020-11-18 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
DK3186376T3 (da) 2014-08-27 2019-05-06 Caribou Biosciences Inc Fremgangsmåder til forbedring af cas9-medieret manipulationseffektivitet
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN108024544B (zh) 2015-07-13 2022-04-29 桑格摩生物治疗股份有限公司 用于核酸酶介导的基因组工程化的递送方法及组合物
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
AU2016343887B2 (en) * 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
JP6976249B2 (ja) 2015-11-23 2021-12-08 サンガモ セラピューティクス, インコーポレイテッド 免疫を工学操作するための方法および組成物
WO2017106537A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the mhc cell receptor
WO2017106528A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the t cell receptor
EP3402533B1 (en) * 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3769775A3 (en) 2016-02-02 2021-03-17 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
CN106397599B (zh) * 2016-02-23 2020-08-07 上海交通大学 二价双特异性抗体杂交蛋白的表达和制备方法
US11471462B2 (en) 2016-06-27 2022-10-18 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
HRP20212025T1 (hr) 2016-08-24 2022-04-01 Sangamo Therapeutics, Inc. Regulacija ekspresije gena pomoću projektiranih nukleaza
WO2018039448A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Engineered target specific nucleases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3509645A4 (en) * 2016-09-07 2020-06-17 Sangamo Therapeutics, Inc. MODULATION OF LIVER GENES
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
MX2019004487A (es) * 2016-10-20 2020-02-07 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.
EP3551169A4 (en) * 2016-12-09 2020-09-09 Sangamo Therapeutics, Inc. TARGET-SPECIFIC NUCLEASES ADMINISTRATION
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3562515A4 (en) * 2016-12-29 2020-11-18 Applied StemCell, Inc. GENE EDITING PROCESS USING A VIRUS
WO2018131551A1 (ja) 2017-01-13 2018-07-19 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 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
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
RU2019139045A (ru) 2017-05-03 2021-06-03 Сангамо Терапьютикс, Инк. Способы и композиции для модификации гена регулятора трансмембранной проводимости при кистозном фиброзе (cftr)
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
CN110997924B (zh) 2017-06-15 2024-12-20 株式会社图尔金 用于在肝中表达感兴趣的蛋白的平台
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
SG11202003464VA (en) * 2017-10-17 2020-05-28 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
WO2019089913A1 (en) * 2017-11-01 2019-05-09 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CA3088180A1 (en) * 2018-01-12 2019-07-18 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
BR112020015488A2 (pt) * 2018-02-08 2020-12-08 Sangamo Therapeutics, Inc. Nucleases alvo-específicas modificadas
US20210130824A1 (en) * 2018-02-16 2021-05-06 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting fibrinogen-alpha
KR20200141470A (ko) 2018-04-06 2020-12-18 칠드런'즈 메디컬 센터 코포레이션 체세포 재프로그래밍 및 각인의 조정을 위한 조성물 및 방법
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108795986A (zh) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 一种a型血友病慢病毒载体、慢病毒及其制备方法和应用
AU2019314513A1 (en) 2018-08-03 2021-01-28 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
CA3106691A1 (en) * 2018-08-07 2020-02-13 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
CA3109592A1 (en) 2018-08-23 2020-02-27 Sangamo Therapeutics, Inc. Engineered target specific base editors
EP3841212A1 (en) * 2018-08-24 2021-06-30 CSL Behring Gene Therapy, Inc. Vector production in serum free media
EP3853244A4 (en) 2018-09-18 2022-09-14 Sangamo Therapeutics, Inc. PROGRAMMED CELL DEATH 1 (PD1) SPECIFIC NUCLEASES
JP2022501036A (ja) * 2018-09-20 2022-01-06 サノフイSanofi イントロンベースの普遍的なクローニング方法および組成物
IL322436A (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
BR112021007229A2 (pt) 2018-10-16 2021-08-10 Blueallele, Llc métodos para inserção dirigida de dna em genes
EP3867377A1 (en) * 2018-10-17 2021-08-25 CRISPR Therapeutics AG Compositions and methods for delivering transgenes
CN113260701A (zh) * 2018-10-18 2021-08-13 英特利亚治疗股份有限公司 用于表达因子ix的组合物和方法
TWI860300B (zh) * 2018-10-18 2024-11-01 美商英特利亞醫療公司 用於從白蛋白基因座表現轉殖基因的組成物及方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020146805A1 (en) 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3826664A4 (en) * 2019-02-06 2022-10-26 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
EP3923992A1 (en) 2019-02-15 2021-12-22 CRISPR Therapeutics AG Gene editing for hemophilia a with improved factor viii expression
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
CN113939586A (zh) 2019-04-02 2022-01-14 桑格摩生物治疗股份有限公司 治疗β-地中海贫血症的方法
KR20250148676A (ko) * 2019-04-03 2025-10-14 리제너론 파마슈티칼스 인코포레이티드 세이프 하버 좌위 내로의 항체 코딩 서열의 삽입을 위한 방법 및 조성물
WO2020210552A1 (en) * 2019-04-11 2020-10-15 California Institute Of Technology Methods and compositions for in vivo gene editing based cell-type-specific cellular engineering
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3141159A1 (en) * 2019-05-21 2020-11-26 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
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
WO2021028359A1 (en) 2019-08-09 2021-02-18 Sangamo Therapeutics France Controlled expression of chimeric antigen receptors in t cells
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021083073A1 (zh) * 2019-10-31 2021-05-06 华东师范大学 一种基于肝细胞Alb基因的疾病治疗的产品
WO2021224633A1 (en) 2020-05-06 2021-11-11 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
BR112023000327A2 (pt) 2020-07-16 2023-01-31 Acuitas Therapeutics Inc Lipídeos catiônicos para o uso em nanopartículas lipídicas
EP4501951A3 (en) 2020-08-25 2025-04-30 Kite Pharma, Inc. T cells with improved functionality
US20240191243A1 (en) * 2021-04-21 2024-06-13 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Hsv amplicon packaging system using engineered cells
MX2024005124A (es) * 2021-10-27 2024-05-16 Regeneron Pharma Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b.
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
WO2023132704A1 (ko) 2022-01-07 2023-07-13 주식회사 툴젠 유전자 편집 과정에서 발생 가능한 오프 타겟을 예측하는 방법
EP4522201A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
US20250302994A1 (en) 2022-05-09 2025-10-02 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease

Family Cites Families (112)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
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
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
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
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2681922C (en) 1994-01-18 2012-05-15 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to 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
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
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
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
AU735763B2 (en) 1997-12-05 2001-07-12 Immune Response Corporation, The Novel vectors and genes exhibiting increased expression
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
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6794136B1 (en) * 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
EP2207032A1 (en) 2000-02-08 2010-07-14 Sangamo BioSciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001259211B2 (en) 2000-04-28 2006-07-13 St. Jude Children's Research Hospital DNA transfection system for the generation of infectious influenza virus
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
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
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
ATE531796T1 (de) 2002-03-21 2011-11-15 Sangamo Biosciences Inc Verfahren und zusammensetzungen zur verwendung von zinkfinger-endonukleasen zur verbesserung der homologen rekombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7238346B2 (en) 2002-07-08 2007-07-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw High capacity recombinant adenoviral vector for treatment of hemophilia A
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP2008506359A (ja) 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
DE602005011943D1 (de) 2004-04-08 2009-02-05 Sangamo Biosciences Inc Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
EP1877583A2 (en) 2005-05-05 2008-01-16 Arizona Board of Regents on behalf of the Unversity of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
KR101419729B1 (ko) 2005-07-26 2014-07-17 상가모 바이오사이언스 인코포레이티드 외래 핵산 서열의 표적화된 통합 및 발현
US20090263900A1 (en) 2008-04-14 2009-10-22 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
AU2006304668B2 (en) 2005-10-18 2013-03-07 Duke University Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
US8129510B2 (en) 2006-03-30 2012-03-06 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
CA2651494C (en) 2006-05-25 2015-09-29 Sangamo Biosciences, Inc. Engineered cleavage half-domains
AU2007267874B2 (en) 2006-05-25 2012-03-15 Sangamo Therapeutics, Inc. Methods and compositions for gene inactivation
WO2008109467A1 (en) 2007-03-02 2008-09-12 Arizona Board Of Regents For And On Behalf Of Arizona State University Electrically conducting porphyrin and porphyrin-fullerene electropolymers
AU2008244473B2 (en) 2007-04-26 2013-06-20 Sangamo Therapeutics, Inc. Targeted integration into the PPP1R12C locus
CN107501370B (zh) 2007-06-19 2021-06-18 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗-反向硫代磷酸类似物的合成和用途
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
WO2009054985A1 (en) 2007-10-25 2009-04-30 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
WO2009130208A1 (en) 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
EP2789691B1 (en) 2008-08-22 2018-08-08 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
JP6215533B2 (ja) 2009-04-09 2017-10-18 サンガモ セラピューティクス, インコーポレイテッド 幹細胞への標的組込み
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3581197A1 (de) 2009-07-31 2019-12-18 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
SG185367A1 (en) 2010-04-26 2012-12-28 Sangamo Biosciences Inc Genome editing of a rosa locus using zinc-finger nucleases
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス, インコーポレイテッド 新規のdna結合タンパク質及びその使用
RU2608643C2 (ru) 2010-10-12 2017-01-23 Дзе Чилдрен`З Хоспитал Оф Филадельфия Способы и композиции для лечения гемофилии в
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US8601579B2 (en) * 2011-06-03 2013-12-03 Apple Inc. System and method for preserving references in sandboxes
JP6208658B2 (ja) 2011-07-05 2017-10-04 バイオアシス テクノロジーズ インコーポレイテッド p97−抗体結合体および使用方法
CA2848417C (en) * 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) * 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
US10570378B2 (en) * 2012-02-28 2020-02-25 Sigma-Aldrich Co. Llc Targeted histone acetylation
WO2013169802A1 (en) * 2012-05-07 2013-11-14 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
ES2613691T3 (es) 2012-07-11 2017-05-25 Sangamo Biosciences, Inc. Métodos y composiciones para el tratamiento de enfermedades por almacenamiento lisosomal
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
HK1223401A1 (zh) 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
CA2931848A1 (en) * 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
US9841814B1 (en) * 2017-01-27 2017-12-12 Emergent AR Platforms Corp. Intentional user experience

Similar Documents

Publication Publication Date Title
JP2017504354A5 (OSRAM)
JP2017500061A5 (OSRAM)
CN108026526B (zh) 用于改善移植的crispr/cas相关方法和组合物
Gao et al. Therapeutic potential of CRISPR/Cas9 gene editing in engineered T‐cell therapy
ES2993268T3 (en) Genetically engineered t cells with regnase-1 and/or tgfbrii disruption have improved functionality and persistence
Huai et al. CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice
JP2018520997A5 (OSRAM)
EP3368689B1 (en) Composition for modulating immune responses by use of immune cell gene signature
JP2018522563A5 (OSRAM)
Wang et al. The clinical applications of genome editing in HIV
JP2018525979A5 (OSRAM)
TW202028466A (zh) 編輯RNAs的方法以及組成物
CN105567735A (zh) 一种凝血因子基因突变的定点修复载体系统及方法
Annoni et al. Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice
JP2013544077A5 (OSRAM)
JP2015533786A5 (OSRAM)
US20190106678A1 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
JP2021502085A5 (OSRAM)
JP2018537087A5 (OSRAM)
WO2017075465A1 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
JP2018531614A5 (OSRAM)
RU2013120986A (ru) Способы и композиции для лечения гемофилии в
HRP20201443T1 (hr) Postupci i pripravci za liječenje genetskog stanja
JP2020500162A5 (OSRAM)
Bueren et al. Advances in the gene therapy of monogenic blood cell diseases