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
JP6642943B2 (ja
JP2017504354A (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 (enExample) 2017-12-21
JP6642943B2 JP6642943B2 (ja) 2020-02-12

Family

ID=53270543

Family Applications (3)

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
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
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 Before (1)

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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) US9771403B2 (enExample)
EP (3) EP3757116A1 (enExample)
JP (3) JP6535684B2 (enExample)
CN (3) CN105874071B (enExample)
AU (3) AU2014363991B2 (enExample)
CA (3) CA2931637C (enExample)
DK (1) DK3080274T3 (enExample)
ES (1) ES2813367T3 (enExample)
HU (1) HUE051628T2 (enExample)
IL (3) IL245778B (enExample)
WO (2) WO2015089046A1 (enExample)

Families Citing this family (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
EP3757116A1 (en) * 2013-12-09 2020-12-30 Sangamo Therapeutics, 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
JP6606088B2 (ja) * 2014-02-24 2019-11-13 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介性標的化組み込みのための方法および組成物
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US9970030B2 (en) 2014-08-27 2018-05-15 Caribou Biosciences, Inc. Methods for increasing CAS9-mediated engineering efficiency
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
WO2017011519A1 (en) 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
MY189674A (en) * 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
AU2016361350B2 (en) 2015-11-23 2023-04-06 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
CN108699132B (zh) 2015-12-18 2023-08-11 桑格摩生物治疗股份有限公司 Mhc细胞受体的靶向破坏
ES2983043T3 (es) 2015-12-18 2024-10-21 Sangamo Therapeutics Inc Alteración dirigida del receptor de células T
EP3402533B1 (en) * 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
US10724020B2 (en) 2016-02-02 2020-07-28 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
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US10975393B2 (en) 2016-08-24 2021-04-13 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
SG11201901531TA (en) * 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Regulation of gene expression using engineered nucleases
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
AU2017345430B2 (en) * 2016-10-20 2024-09-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
CA3045122A1 (en) * 2016-12-09 2018-06-14 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
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
US20230190958A1 (en) * 2017-01-13 2023-06-22 Jichi Medical University AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome
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
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11820728B2 (en) 2017-04-28 2023-11-21 Acuitas Therapeutics, Inc. Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN110869497A (zh) 2017-05-03 2020-03-06 桑格摩生物治疗股份有限公司 修饰囊性纤维化跨膜传导调节蛋白(cftr)基因的方法和组合物
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
AU2018283686A1 (en) 2017-06-15 2020-01-30 Toolgen Incorporated Platform for expressing protein of interest in liver
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
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
EP3697907A1 (en) * 2017-10-17 2020-08-26 CRISPR Therapeutics AG Compositions and methods for gene editing for hemophilia a
US20200299658A1 (en) * 2017-11-01 2020-09-24 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
EP3737762A1 (en) 2018-01-12 2020-11-18 CRISPR Therapeutics AG Compositions and methods for gene editing by targeting transferrin
JP7275152B2 (ja) * 2018-02-08 2023-05-17 サンガモ セラピューティクス, インコーポレイテッド 操作された標的特異的なヌクレアーゼ
US20210130824A1 (en) * 2018-02-16 2021-05-06 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting fibrinogen-alpha
WO2019195738A1 (en) 2018-04-06 2019-10-10 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2019226953A1 (en) 2018-05-23 2019-11-28 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
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス, インコーポレイテッド 操作された標的特異的な塩基エディター
SG11202101594WA (en) * 2018-08-24 2021-03-30 Csl Behring Gene Therapy Inc Vector production in serum free media
WO2020061161A1 (en) 2018-09-18 2020-03-26 Sangamo Therapeutics, Inc. Programmed cell death 1 (pd1) specific nucleases
CN113015801A (zh) * 2018-09-20 2021-06-22 赛诺菲 基于内含子的通用克隆方法和组合物
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
GB2611929B (en) 2018-10-16 2023-11-22 Blueallele Corp Methods for targeted insertion of DNA in genes
JP7520826B2 (ja) * 2018-10-17 2024-07-23 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 導入遺伝子を送達するための組成物および方法
EP3867381A2 (en) * 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
BR112021007301A2 (pt) * 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US11453639B2 (en) 2019-01-11 2022-09-27 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
JP2022519410A (ja) * 2019-02-06 2022-03-24 サンガモ セラピューティクス, インコーポレイテッド ムコ多糖症i型の処置のための方法
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
CA3132167A1 (en) 2019-04-02 2020-10-08 Weston P. MILLER IV Methods for the treatment of beta-thalassemia
CN118064502A (zh) * 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
US11702644B2 (en) * 2019-04-11 2023-07-18 California Institute Of Technology Methods and compositions for in vivo gene editing based cell-type-specific cellular engineering
US12473543B2 (en) 2019-04-17 2025-11-18 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
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
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
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
MX2023000614A (es) 2020-07-16 2023-02-13 Acuitas Therapeutics Inc Lipidos cationicos para usarse en nanoparticulas lipidicas.
US12152251B2 (en) 2020-08-25 2024-11-26 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
EP4423119A1 (en) * 2021-10-27 2024-09-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
IL313486A (en) 2021-12-16 2024-08-01 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
AU2023205708A1 (en) 2022-01-07 2024-07-25 Toolgen Incorporated Method for predicting possible off-targets in gene editing process
EP4522203A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
EP4522201A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
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
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (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
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The 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
US6140466A (en) 1994-01-18 2000-10-31 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
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
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
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
WO1999045132A1 (en) 1998-03-02 1999-09-10 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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
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
AU776576B2 (en) * 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
JP5047437B2 (ja) 2000-02-08 2012-10-10 サンガモ バイオサイエンシーズ, インコーポレイテッド 薬物の発見のための細胞
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
DE60137345D1 (de) 2000-04-28 2009-02-26 St Jude Childrens Res Hospital Dna-transfektionssystem zur erzeugung von infektiösen negativsträngigen rna virus
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology 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
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
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
CA2392863A1 (en) 2002-07-08 2004-01-08 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A 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
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
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
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
ES2315859T3 (es) 2004-04-08 2009-04-01 Sangamo Biosciences, Inc. Metodos y composiciones para tratar afecciones neuropaticas y neurodegenerativas.
AU2005287278B2 (en) 2004-09-16 2011-08-04 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
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
ES2626025T3 (es) 2005-10-18 2017-07-21 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
US8129510B2 (en) 2006-03-30 2012-03-06 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
EP2213731B1 (en) 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
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
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
SI2167523T1 (sl) 2007-06-19 2014-09-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Sinteza in uporaba anti-reverznih fosforotioatnih analogov kape obveščevalne RNA
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
AU2008317354B2 (en) 2007-10-25 2014-04-10 Ospedale San Raffaele S.R.L. Methods and compositions for targeted integration
WO2009131632A1 (en) 2008-04-14 2009-10-29 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
CN102625655B (zh) 2008-12-04 2016-07-06 桑格摩生物科学股份有限公司 使用锌指核酸酶在大鼠中进行基因组编辑
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US9834787B2 (en) 2009-04-09 2017-12-05 Sangamo Therapeutics, Inc. Targeted integration into stem cells
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
DK2459231T3 (en) 2009-07-31 2016-09-05 Ethris Gmbh RNA with a combination of unmodified and modified nucleotides for protein expression
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
WO2012051343A1 (en) * 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
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
BR112014002219A2 (pt) 2011-07-05 2018-08-07 Bioasis Technologies Inc conjugados anticorpo p97 e métodos de sua utilização
CA3186126A1 (en) * 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
CN105658792B (zh) * 2012-02-28 2020-11-10 西格马-奥尔德里奇有限责任公司 靶向组蛋白乙酰化
BR112014027813A2 (pt) * 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
ES2697912T3 (es) 2012-07-11 2019-01-29 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de enfermedades monogénicas
CA2888931C (en) 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
EP3757116A1 (en) * 2013-12-09 2020-12-30 Sangamo Therapeutics, 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 (enExample)
JP2017500061A5 (enExample)
CN108026526B (zh) 用于改善移植的crispr/cas相关方法和组合物
ES2993268T3 (en) Genetically engineered t cells with regnase-1 and/or tgfbrii disruption have improved functionality and persistence
ChuahMarinee Transposons: moving forward from preclinical studies to clinical trials
Huai et al. CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice
JP2018520997A5 (enExample)
EP3368689B1 (en) Composition for modulating immune responses by use of immune cell gene signature
JP2018522563A5 (enExample)
Wang et al. The clinical applications of genome editing in HIV
JP2018525979A5 (enExample)
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 (enExample)
JP2015533786A5 (enExample)
JP2021502085A5 (enExample)
JP2018537087A5 (enExample)
WO2017075465A1 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
RU2013120986A (ru) Способы и композиции для лечения гемофилии в
JP2017538401A5 (enExample)
HRP20201443T1 (hr) Postupci i pripravci za liječenje genetskog stanja
JP2020500162A5 (enExample)
Bueren et al. Advances in the gene therapy of monogenic blood cell diseases
JP2013530689A5 (enExample)
JP2020505044A5 (enExample)