JP2014523748A5 - - Google Patents

Download PDF

Info

Publication number
JP2014523748A5
JP2014523748A5 JP2014522973A JP2014522973A JP2014523748A5 JP 2014523748 A5 JP2014523748 A5 JP 2014523748A5 JP 2014522973 A JP2014522973 A JP 2014522973A JP 2014522973 A JP2014522973 A JP 2014522973A JP 2014523748 A5 JP2014523748 A5 JP 2014523748A5
Authority
JP
Japan
Prior art keywords
composition
protein
stem cells
cell
cells
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
JP2014522973A
Other languages
English (en)
Japanese (ja)
Other versions
JP6072788B2 (ja
JP2014523748A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2012/048176 external-priority patent/WO2013016446A2/en
Publication of JP2014523748A publication Critical patent/JP2014523748A/ja
Publication of JP2014523748A5 publication Critical patent/JP2014523748A5/ja
Application granted granted Critical
Publication of JP6072788B2 publication Critical patent/JP6072788B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2014522973A 2011-07-25 2012-07-25 嚢胞性線維症膜コンダクタンス制御因子（ｃｆｔｒ）遺伝子を改変するための方法および組成物 Expired - Fee Related JP6072788B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161511434P 2011-07-25 2011-07-25
US61/511,434 2011-07-25
PCT/US2012/048176 WO2013016446A2 (en) 2011-07-25 2012-07-25 Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene

Publications (3)

Publication Number Publication Date
JP2014523748A JP2014523748A (ja) 2014-09-18
JP2014523748A5 true JP2014523748A5 (enExample) 2015-07-16
JP6072788B2 JP6072788B2 (ja) 2017-02-01

Family

ID=47601755

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2014522973A Expired - Fee Related JP6072788B2 (ja) 2011-07-25 2012-07-25 嚢胞性線維症膜コンダクタンス制御因子（ｃｆｔｒ）遺伝子を改変するための方法および組成物

Country Status (6)

Country Link
US (2) US9161995B2 (enExample)
EP (1) EP2737063B1 (enExample)
JP (1) JP6072788B2 (enExample)
AU (1) AU2012286901B2 (enExample)
CA (1) CA2841541C (enExample)
WO (1) WO2013016446A2 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105518146B (zh) 2013-04-04 2022-07-15 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
US11306328B2 (en) * 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
WO2015179656A2 (en) * 2014-05-23 2015-11-26 The Scripps Research Institute Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr)
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
HK1254190A1 (zh) 2015-05-08 2019-07-12 President And Fellows Of Harvard College 通用供体干细胞和相关方法
CA2984013A1 (en) * 2015-05-12 2016-11-17 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2018224843B2 (en) 2017-02-27 2024-08-15 Translate Bio, Inc. Methods for purification of messenger RNA
US11655275B2 (en) 2017-05-03 2023-05-23 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
JP2022516661A (ja) * 2019-01-08 2022-03-01 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 嚢胞性線維症を処置する方法
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia Therapeutics, Inc. Methods and compositions for directed genome editing

Family Cites Families (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
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
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
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
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to binding proteins for recognition of dna
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5948681A (en) * 1996-08-14 1999-09-07 Children's Hospital Of Philadelphia Non-viral vehicles for use in gene transfer
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
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
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
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells 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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
CA2398590C (en) 2000-02-08 2012-08-28 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
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
CA2474486C (en) 2002-01-23 2013-05-14 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
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
JP4966006B2 (ja) * 2003-01-28 2012-07-04 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
DK2927318T3 (da) 2003-08-08 2020-08-03 Sangamo Therapeutics Inc Fremgangsmåde og sammensætninger til målrettet spaltning og rekombination
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
JP4903689B2 (ja) 2004-04-08 2012-03-28 サンガモ バイオサイエンシズ インコーポレイテッド 神経障害および神経変性症状を治療するための方法と組成物
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
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
WO2006121866A2 (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
WO2007014181A2 (en) 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
CA3055968A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
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
CA2700231C (en) 2007-09-27 2018-09-18 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
WO2009100373A1 (en) 2008-02-08 2009-08-13 Sangamo Biosciences, Inc. Treatment of chronic pain with zinc finger proteins
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
WO2009146179A1 (en) * 2008-04-15 2009-12-03 University Of Iowa Research Foundation Zinc finger nuclease for the cftr gene and methods of use thereof
EP2128245A1 (en) 2008-05-27 2009-12-02 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Generation of induced pluripotent stem (iPS) cells
EP2313515B1 (en) 2008-08-22 2015-03-04 Sangamo BioSciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
US20110023151A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of abc transporters
US20100158868A1 (en) 2008-12-19 2010-06-24 Yuet W. Kan Use of Fetal Cells for the Treatment of Genetic Diseases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
CN102812122A (zh) 2009-05-27 2012-12-05 索尔克生物学研究院 基因矫正的无疾病的诱导的多能干细胞的产生
AU2010327998B2 (en) 2009-12-10 2015-11-12 Iowa State University Research Foundation, Inc. TAL effector-mediated DNA modification
WO2011097036A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
CA2993567C (en) * 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene

Similar Documents

Publication Publication Date Title
JP2014523748A5 (enExample)
JP2021502085A5 (enExample)
SG178940A1 (en) Products and methods for enhanced transgene expression and processing
MX2015012361A (es) Metodos de fabricacion para controlar el contenido de lisina c-terminal, galactosa y acido sialico en proteinas recombinantes.
IN2014CN01706A (enExample)
GEP201606544B (en) Excision of transgenes in genetically modified organisms
ATE419336T1 (de) Verfahren und materialien zur herstellung organischer produkte in zellen von candida- spezies
RU2019114217A (ru) Геномная инженерия
JP2010500029A5 (enExample)
EA201300054A1 (ru) Полипептид, обладающий активностью бета-глюкозидазы, и его применение
WO2016048995A3 (en) Fgf19 truncations and mutants and uses thereof
PH12013501592A1 (en) Novel use of a dense and erect panicle 1 gene in improving nitrogen utilization efficiency
WO2010042749A3 (en) Chimeric therapeutics, compositions, and methods for using same
MY161859A (en) Personalized production of biologics and method for reprogramming somatic cells
MX365095B (es) Métodos y composiciones para incrementar la producción de ácido siálico y tratar condiciones de enfermedades relacionadas siálicas.
EA201300056A1 (ru) Полипептид с активностью ацетилксиланэстеразы и его применения
WO2010042751A3 (en) Chimeric therapeutics, compositions, and methods for using same
MX2015015274A (es) Ruta de ácido organico novedosa.
MY180183A (en) Methods and compositions for preventing norleucine misincorporation into proteins
IN2014DN00094A (enExample)
AU2015218647A1 (en) Diacylglycerol acyl transferase (DGA1) polynucleotides, and methods of increasing yeast cell lipid production by overexpression of heterologous DGA1
ATE546464T1 (de) Verfahren zur produktion von rekombinantem protein in cho-zellen
WO2013052814A3 (en) E1 enzyme mutants and uses thereof
EA201300059A1 (ru) Полипептид, обладающий активностью сволленина, и его применение
Gokhale et al. Strategy abundance in evolutionary many-player games with multiple strategies