CN105934524A - 用于治疗亨廷顿氏病的方法和组合物 - Google Patents

用于治疗亨廷顿氏病的方法和组合物 Download PDF

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CN105934524A
CN105934524A CN201480072869.5A CN201480072869A CN105934524A CN 105934524 A CN105934524 A CN 105934524A CN 201480072869 A CN201480072869 A CN 201480072869A CN 105934524 A CN105934524 A CN 105934524A
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htt
zfp
domain
cells
protein
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H·S·张
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Sangamo Therapeutics Inc
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Sangamo Biosciences Inc
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CN201480072869.5A 2013-11-11 2014-11-11 用于治疗亨廷顿氏病的方法和组合物 Pending CN105934524A (zh)

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Application Number Priority Date Filing Date Title
US201361902704P 2013-11-11 2013-11-11
US61/902,704 2013-11-11
PCT/US2014/064987 WO2015070212A1 (en) 2013-11-11 2014-11-11 Methods and compositions for treating huntington's disease

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CN105934524A true CN105934524A (zh) 2016-09-07

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US (1) US10369201B2 (enExample)
EP (1) EP3068905A4 (enExample)
JP (2) JP2016537341A (enExample)
KR (1) KR102431079B1 (enExample)
CN (1) CN105934524A (enExample)
AU (1) AU2014346424B2 (enExample)
BR (1) BR112016010175A2 (enExample)
CA (1) CA2929179A1 (enExample)
MX (1) MX391904B (enExample)
NZ (1) NZ719477A (enExample)
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Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106636388A (zh) * 2016-12-15 2017-05-10 湖南中能荆卫生物科技有限公司 用于亨廷顿病诊断的基因及其应用以及由其编码的蛋白和蛋白的应用
CN109112162A (zh) * 2018-08-30 2019-01-01 西南大学 利用CRISPR/Cas9技术构建的亨廷顿病原位敲入小鼠模型及构建方法
CN110035776A (zh) * 2016-09-22 2019-07-19 马萨诸塞大学 亨廷顿病的aav治疗
CN110747208A (zh) * 2019-11-28 2020-02-04 海南大学 一种木薯硝酸还原酶基因及其过量表达载体的构建和抗病应用
CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi
CN112313246A (zh) * 2018-04-18 2021-02-02 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
CN112839709A (zh) * 2018-06-06 2021-05-25 加利福尼亚大学董事会 治疗神经退行性疾病的神经干细胞组合物和方法
CN113301909A (zh) * 2019-01-15 2021-08-24 桑格摩生物治疗股份有限公司 Htt阻遏物及其应用
CN114728018A (zh) * 2019-11-01 2022-07-08 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
CN115786395A (zh) * 2022-08-19 2023-03-14 南京启真基因工程有限公司 构建htt基因突变的亨廷顿舞蹈症模型猪核移植供体细胞的基因编辑系统及其应用

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
IN2012DN00720A (enExample) 2009-07-06 2015-06-19 Ontorii Inc
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
MX347361B (es) 2011-07-19 2017-04-12 Wave Life Sciences Ltd Metodos para la sintesis de acidos nucleicos funcionalizados.
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
AU2013287630B2 (en) 2012-07-13 2017-05-25 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
US20160362667A1 (en) * 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018081806A2 (en) * 2016-10-31 2018-05-03 University Of Florida Research Foundation, Inc. Compositions and methods for impeding transcription of expanded microsatellite repeats
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
JP7381476B2 (ja) * 2017-10-24 2023-11-15 サンガモ セラピューティクス， インコーポレイテッド 希少疾患の処置のための方法および組成物
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
BR112021020868A2 (pt) 2019-04-23 2021-12-14 Sangamo Therapeutics Inc Moduladores da expressão do gene quadro aberto de leitura 72 do cromossomo 9 e seus usos
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021087358A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Gin recombinase variants
US20230340470A1 (en) * 2019-12-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Methods for treating huntington's disease
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
IL321780A (en) * 2023-01-05 2025-08-01 Emugen Therapeutics Llc Neural promoters and their uses
WO2024148179A2 (en) * 2023-01-05 2024-07-11 Emugen Therapeutics Llc Retro-aav and use in treating neurodegenerative diseases
TW202515898A (zh) 2023-06-30 2025-04-16 日商武田藥品工業股份有限公司 Ｈｔｔ抑制物及其用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070060606A1 (en) * 1999-10-07 2007-03-15 Robertson Harold A Compounds and methods for modulating phosphodiesterase 10A
US20100299768A1 (en) * 2007-06-18 2010-11-25 Commissariat A L'energie Atomique Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and its Application for the Treatment of Huntington's Disease
US20110082093A1 (en) * 2009-07-28 2011-04-07 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
WO2013130824A1 (en) * 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease

Family Cites Families (100)

* 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
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
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
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
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
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
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
US6242568B1 (en) 1994-01-18 2001-06-05 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
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
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
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
DE69535829D1 (de) 1994-08-20 2008-10-16 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der 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
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for 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
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
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
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
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
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
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
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
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
AU5587001A (en) 2000-04-28 2001-11-12 Sangamo Biosciences Inc Databases of regulatory sequences; methods of making and using same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
IL154059A0 (en) 2000-07-21 2003-07-31 Syngenta Participations Ag Zinc finger domain recognition code and uses thereof
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
ATE360361T1 (de) 2000-09-28 2007-05-15 Sangamo Biosciences Inc Kernumprogrammierung bei iswi und ähnlichen chromatinremodellierenden atpasen
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
RU2192884C2 (ru) * 2000-11-09 2002-11-20 Государственное учреждение Научно-исследовательский институт клинической иммунологии СО РАМН Вакцина для стимуляции противоопухолевого иммунитета
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
EP1421177A4 (en) 2001-08-20 2006-06-07 Scripps Research Inst ZINC FINGER FASTENING 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
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
JP2005520519A (ja) 2002-03-15 2005-07-14 セレクティス ハイブリッドおよび単鎖メガヌクレアーゼならびにその使用
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. 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
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
AU2004208031B2 (en) 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
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
WO2005099771A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
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
DK2628794T3 (en) 2005-10-18 2016-08-15 Prec Biosciences RATIONALE CONSTRUCTED MECHANUCLEAS WITH CHANGED SEQUENCE SPECIFICITY AND DNA BINDING EFFICIENCY
US7985778B2 (en) 2006-03-07 2011-07-26 Koichi Nakamura Method for decomposing and reclaiming synthetic resin having ester bond in the composition structure
JP2009537140A (ja) 2006-05-19 2009-10-29 サンガモ バイオサイエンシーズ， インコーポレイテッド ジヒドロ葉酸還元酵素の不活性化のための方法及び組成物
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝子不活性化のための方法と組成物
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
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
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
JP2010540534A (ja) 2007-09-28 2010-12-24 イントレキソン コーポレーション 生体治療分子の発現のための治療遺伝子スイッチ構築物およびバイオリアクター、ならびにその使用
KR101802393B1 (ko) 2008-06-10 2017-11-28 상가모 테라퓨틱스, 인코포레이티드 Ｂａｘ- 및 ｂａｋ-결함 세포주들의 제조 방법 및 조성물
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
ES2587963T3 (es) 2009-07-31 2016-10-27 Ethris Gmbh ARN con una combinación de nucleótidos no modificados y modificados para la expresión de proteínas
EP2534173B1 (en) 2010-02-08 2019-09-11 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
CN102939377B (zh) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 使用锌指核酸酶对Rosa位点进行基因组编辑
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
ES2713560T3 (es) 2010-10-15 2019-05-22 Fund Centre De Regulacio Genòmica Péptidos que tienen dominios de dedos de zinc y usos de estos
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
CA2854819C (en) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070060606A1 (en) * 1999-10-07 2007-03-15 Robertson Harold A Compounds and methods for modulating phosphodiesterase 10A
US20100299768A1 (en) * 2007-06-18 2010-11-25 Commissariat A L'energie Atomique Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and its Application for the Treatment of Huntington's Disease
US20110082093A1 (en) * 2009-07-28 2011-04-07 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
WO2013130824A1 (en) * 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
A.L.O.HEBB等: "STRIATAL PHOSPHODIESTERASE mRNA AND PROTEIN LEVELS AREREDUCED IN HUNTINGTON_S DISEASE TRANSGENIC MICE PRIORTO THE ONSET OF MOTOR SYMPTOMS", 《NEUROSCIENCE》 *
JANG-HO J. CHA等: "Altered brain neurotransmitter receptors in transgenic miceexpressing a portion of an abnormal human Huntingtondisease gene", 《PROC. NATL. ACAD. SCI》 *
STANEK LISA等: "antisense oligonuleotide-mediated correction of transcriptional dysregulations is correlated with behavioral benefits in the YAC128 mouse model of huntington"s disease", 《JOURNAL OF HUNTINGTON‘S DISEASE》 *
叶翠芳等: "R6/2型亨廷顿病转基因小鼠胰岛β细胞功能损伤", 《中国病理生理杂志》 *

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* Cited by examiner, † Cited by third party
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CN110035776B (zh) * 2016-09-22 2023-08-25 马萨诸塞大学 亨廷顿病的aav治疗
CN110035776A (zh) * 2016-09-22 2019-07-19 马萨诸塞大学 亨廷顿病的aav治疗
CN106636388A (zh) * 2016-12-15 2017-05-10 湖南中能荆卫生物科技有限公司 用于亨廷顿病诊断的基因及其应用以及由其编码的蛋白和蛋白的应用
US12331296B2 (en) 2017-09-22 2025-06-17 Genzyme Corporation Variant RNAi
CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
CN112313246A (zh) * 2018-04-18 2021-02-02 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
CN112313246B (zh) * 2018-04-18 2025-05-13 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
CN112839709A (zh) * 2018-06-06 2021-05-25 加利福尼亚大学董事会 治疗神经退行性疾病的神经干细胞组合物和方法
CN109112162A (zh) * 2018-08-30 2019-01-01 西南大学 利用CRISPR/Cas9技术构建的亨廷顿病原位敲入小鼠模型及构建方法
CN113301909A (zh) * 2019-01-15 2021-08-24 桑格摩生物治疗股份有限公司 Htt阻遏物及其应用
CN114728018A (zh) * 2019-11-01 2022-07-08 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
CN110747208B (zh) * 2019-11-28 2022-04-22 海南大学 一种木薯硝酸还原酶基因及其过量表达载体的构建和抗病应用
CN110747208A (zh) * 2019-11-28 2020-02-04 海南大学 一种木薯硝酸还原酶基因及其过量表达载体的构建和抗病应用
CN115786395A (zh) * 2022-08-19 2023-03-14 南京启真基因工程有限公司 构建htt基因突变的亨廷顿舞蹈症模型猪核移植供体细胞的基因编辑系统及其应用

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