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
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
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
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
SG10202112057QA (en) * 2015-05-12 2021-12-30 Sangamo Therapeutics Inc Nuclease-mediated regulation of gene expression
ES2899323T3 (es) * 2017-02-27 2022-03-10 Translate Bio Inc Métodos de purificación de ARN mensajero
JP2020518245A (ja) * 2017-05-03 2020-06-25 サンガモ セラピューティクス， インコーポレイテッド 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
US20220152221A1 (en) * 2019-01-08 2022-05-19 Asklepios Biopharmaceutical, Inc. A method of treating cystic fibrosis
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite 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
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
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
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
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
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
JP4012243B2 (ja) 1994-01-18 2007-11-21 ザ スクリップス リサーチ インスティチュート 亜鉛フィンガータンパク質誘導体およびそのための方法
CA2186118C (en) 1994-03-23 2010-10-19 Richard W. Hanson Compacted nucleic acids and their delivery to cells
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo 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
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
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
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
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
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
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
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
US7013219B2 (en) 1999-01-12 2006-03-14 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
AU2001250774B2 (en) 2000-02-08 2005-01-27 Sangamo Biosciences, Inc. Cells expressing zinc finger proteins for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 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
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F 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
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, 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
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US20060206949A1 (en) * 2003-01-28 2006-09-14 Sylvain Arnould Custom-made meganuclease and use thereof
EP2927318B1 (en) * 2003-08-08 2020-05-20 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
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
ATE418346T1 (de) 2004-04-08 2009-01-15 Sangamo Biosciences Inc Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
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
US20100055793A1 (en) * 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
EP1913149A4 (en) 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
DK3246403T3 (da) 2005-10-18 2020-10-26 Prec Biosciences Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
JP5763341B2 (ja) 2007-09-27 2015-08-12 サンガモ バイオサイエンシーズ， インコーポレイテッド 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法
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
AU2009212247A1 (en) * 2008-02-08 2009-08-13 Sangamo Therapeutics, Inc. Treatment of chronic pain with zinc finger proteins
CA2720903C (en) 2008-04-14 2019-01-15 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
JP5908725B2 (ja) 2008-08-22 2016-04-26 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的一本鎖開裂および標的組込みのための方法、並びに組成物
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
WO2010138750A2 (en) 2009-05-27 2010-12-02 The Salk Institute For Biological Studies Generation of genetically corrected disease-free induced pluripotent stem cells
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
JP6137596B2 (ja) 2010-02-08 2017-05-31 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作された切断ハーフドメイン
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
EP2566972B1 (en) 2010-05-03 2020-01-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
CA2805442C (en) * 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus

Similar Documents

Publication Publication Date Title
JP2021502085A5 (enExample)
EA201001435A1 (ru) Клетка, ферментирующая сахар-пентозу
UA106193C2 (ru) Гомологичная рекомбинация, опосредованная нуклеазами с цинковыми пальцами
MY172423A (en) Isopropylmalate synthase from nicotiana tabacum and methods and uses thereof
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.
EA201490703A1 (ru) Белковые каркасы на основе фибронектинового повтора типа iii с альтернативными поверхностями связывания
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
MY161859A (en) Personalized production of biologics and method for reprogramming somatic cells
PH12013501592A1 (en) Novel use of a dense and erect panicle 1 gene in improving nitrogen utilization efficiency
WO2010042755A3 (en) Chimeric therapeutics, compositions, and methods for using same
WO2013016724A3 (en) Decarboxylase proteins with high keto-isovalerate decarboxylase activity
WO2010042749A3 (en) Chimeric therapeutics, compositions, and methods for using same
WO2012106465A3 (en) Methods and compositions for increasing sialic acid production and treating sialic related disease conditions
EA201300056A1 (ru) Полипептид с активностью ацетилксиланэстеразы и его применения
MY180183A (en) Methods and compositions for preventing norleucine misincorporation into proteins
IN2014DN00094A (enExample)
ATE546464T1 (de) Verfahren zur produktion von rekombinantem protein in cho-zellen
WO2013052814A8 (en) E1 enzyme mutants and uses thereof
JP2022033728A (ja) 改変細胞、調製方法、及び構築物
MX2016007109A (es) Proteina estructurante de hielo.