KR20220003561A - 염색체 9 오픈 리딩 프레임 72 유전자 발현의 조정제 및 그의 용도 - Google Patents

염색체 9 오픈 리딩 프레임 72 유전자 발현의 조정제 및 그의 용도 Download PDF

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KR20220003561A
KR20220003561A KR1020217037624A KR20217037624A KR20220003561A KR 20220003561 A KR20220003561 A KR 20220003561A KR 1020217037624 A KR1020217037624 A KR 1020217037624A KR 20217037624 A KR20217037624 A KR 20217037624A KR 20220003561 A KR20220003561 A KR 20220003561A
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zfp
c9orf72
seq
fusion protein
cell
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조셉 에프. 나반
암루타 파타마타
모함마드 사미에
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상가모 테라퓨틱스, 인코포레이티드
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KR1020217037624A 2019-04-23 2020-04-23 염색체 9 오픈 리딩 프레임 72 유전자 발현의 조정제 및 그의 용도 Pending KR20220003561A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962837523P 2019-04-23 2019-04-23
US62/837,523 2019-04-23
US202062964844P 2020-01-23 2020-01-23
US62/964,844 2020-01-23
PCT/US2020/029590 WO2020219726A1 (en) 2019-04-23 2020-04-23 Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof

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KR20220003561A true KR20220003561A (ko) 2022-01-10

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US (3) US12139517B2 (https=)
EP (1) EP3958871A1 (https=)
JP (2) JP7618581B2 (https=)
KR (1) KR20220003561A (https=)
CN (2) CN113766921B (https=)
AU (1) AU2020262281B2 (https=)
BR (1) BR112021020868A2 (https=)
CA (1) CA3137368A1 (https=)
CO (1) CO2021015653A2 (https=)
IL (1) IL287384A (https=)
MX (1) MX2021012836A (https=)
PE (1) PE20212332A1 (https=)
PH (1) PH12021552554A1 (https=)
SG (1) SG11202111443SA (https=)
TW (2) TW202521561A (https=)
WO (1) WO2020219726A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3120799A1 (en) 2018-12-20 2020-06-25 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
MX2021012836A (es) * 2019-04-23 2021-12-10 Sangamo Therapeutics Inc Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos.
GB202010075D0 (en) * 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
JP2024534523A (ja) 2021-09-21 2024-09-20 スクライブ・セラピューティクス・インコーポレイテッド 操作されたcasxリプレッサー系

Family Cites Families (76)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
JP4285766B2 (ja) 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von 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
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
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
DE69929600T2 (de) 1998-05-27 2006-09-07 Avigen Inc., Alameda Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
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
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
WO2001025255A2 (en) 1999-10-07 2001-04-12 Baylor College Of Medicine Novel zinc finger protein
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
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
AU9485101A (en) 2000-09-28 2002-04-08 Sangamo Biosciences Inc Nuclear reprogramming using iwsi and related chromatin remodeling atpases
US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US7182944B2 (en) 2001-04-25 2007-02-27 The United States Of America As Represented By The Department Of Health And Human Services Methods of increasing distribution of nucleic acids
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
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
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
US20060239966A1 (en) 2003-10-20 2006-10-26 Tornoee Jens In vivo gene therapy of parkinson's disease
PT1807009E (pt) 2004-10-05 2015-02-25 Univ California Cânula escalonada
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
AU2006283189B2 (en) 2005-08-23 2013-01-31 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
WO2007127803A2 (en) 2006-04-25 2007-11-08 The Regents Of The University Of California Administration of growth factors for the treatment of cns disorders
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
US7837668B2 (en) 2006-10-10 2010-11-23 Ceregene, Inc. Needle assembly for use in delivering precise dosages of proteinaceous pharmaceutical compositions and methods for use of same
WO2008157688A2 (en) 2007-06-19 2008-12-24 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap
CA2700231C (en) 2007-09-27 2018-09-18 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
AU2009212247A1 (en) 2008-02-08 2009-08-13 Sangamo Therapeutics, Inc. Treatment of chronic pain with zinc finger proteins
KR101802393B1 (ko) 2008-06-10 2017-11-28 상가모 테라퓨틱스, 인코포레이티드 Bax- 및 bak-결함 세포주들의 제조 방법 및 조성물
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
JP5866283B2 (ja) 2009-07-28 2016-02-17 サンガモ バイオサイエンシーズ, インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP3594333B1 (en) 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
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
AU2012340213B2 (en) 2011-11-16 2017-12-07 Sangamo Therapeutics, Inc. Modified DNA-binding proteins and uses thereof
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
AU2014223344A1 (en) 2013-02-27 2015-10-01 Massachusetts Institute Of Technology T cell balance gene expression, compositions of matters and methods of use thereof
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
WO2015070212A1 (en) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
WO2015153760A2 (en) 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
GB201418965D0 (https=) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
WO2017040813A2 (en) 2015-09-02 2017-03-09 University Of Massachusetts Detection of gene loci with crispr arrayed repeats and/or polychromatic single guide ribonucleic acids
JP2019500899A (ja) 2015-11-23 2019-01-17 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア CRISPR/Cas9の核送達を通じた細胞RNAの追跡と操作
BR112018012894A2 (en) 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
WO2018002762A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
MA46018A (fr) 2016-08-19 2019-06-26 Bluebird Bio Inc Activateurs d'édition du génome
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
WO2018039471A2 (en) 2016-08-25 2018-03-01 Trustees Of Boston University Synthetic transcriptional and epigenetic regulators based on engineered, orthogonal zinc finger proteins
CN110214184B (zh) 2016-12-01 2024-07-02 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
CA3079727A1 (en) 2017-10-24 2019-05-02 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
MX2021012836A (es) * 2019-04-23 2021-12-10 Sangamo Therapeutics Inc Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos.

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