CN108348576B - Htt阻抑物及其用途 - Google Patents

Htt阻抑物及其用途 Download PDF

Info

Publication number
CN108348576B
CN108348576B CN201680061809.2A CN201680061809A CN108348576B CN 108348576 B CN108348576 B CN 108348576B CN 201680061809 A CN201680061809 A CN 201680061809A CN 108348576 B CN108348576 B CN 108348576B
Authority
CN
China
Prior art keywords
htt
cell
cells
protein
domain
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.)
Active
Application number
CN201680061809.2A
Other languages
English (en)
Chinese (zh)
Other versions
CN108348576A (zh
Inventor
S·弗洛里奇
J·C·米勒
D·帕斯乔恩
E·J·瑞巴
B·泽特勒
H·S·张
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of CN108348576A publication Critical patent/CN108348576A/zh
Application granted granted Critical
Publication of CN108348576B publication Critical patent/CN108348576B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0656—Adult fibroblasts
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Rheumatology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CN201680061809.2A 2015-09-23 2016-09-23 Htt阻抑物及其用途 Active CN108348576B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562222588P 2015-09-23 2015-09-23
US62/222,588 2015-09-23
PCT/US2016/053374 WO2017053753A1 (en) 2015-09-23 2016-09-23 Htt repressors and uses thereof

Publications (2)

Publication Number Publication Date
CN108348576A CN108348576A (zh) 2018-07-31
CN108348576B true CN108348576B (zh) 2022-01-11

Family

ID=58387548

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680061809.2A Active CN108348576B (zh) 2015-09-23 2016-09-23 Htt阻抑物及其用途

Country Status (23)

Country Link
US (3) US10435441B2 (cg-RX-API-DMAC7.html)
EP (1) EP3352776B1 (cg-RX-API-DMAC7.html)
JP (2) JP6853257B2 (cg-RX-API-DMAC7.html)
KR (1) KR102803519B1 (cg-RX-API-DMAC7.html)
CN (1) CN108348576B (cg-RX-API-DMAC7.html)
AU (2) AU2016325613B2 (cg-RX-API-DMAC7.html)
BR (1) BR112018005779A2 (cg-RX-API-DMAC7.html)
CL (1) CL2018000749A1 (cg-RX-API-DMAC7.html)
DK (1) DK3352776T3 (cg-RX-API-DMAC7.html)
EA (1) EA037993B1 (cg-RX-API-DMAC7.html)
ES (1) ES3032775T3 (cg-RX-API-DMAC7.html)
FI (1) FI3352776T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20250728T1 (cg-RX-API-DMAC7.html)
HU (1) HUE071655T2 (cg-RX-API-DMAC7.html)
IL (1) IL258030B (cg-RX-API-DMAC7.html)
LT (1) LT3352776T (cg-RX-API-DMAC7.html)
MX (1) MX391052B (cg-RX-API-DMAC7.html)
PL (1) PL3352776T3 (cg-RX-API-DMAC7.html)
PT (1) PT3352776T (cg-RX-API-DMAC7.html)
RS (1) RS66924B1 (cg-RX-API-DMAC7.html)
SI (1) SI3352776T1 (cg-RX-API-DMAC7.html)
WO (1) WO2017053753A1 (cg-RX-API-DMAC7.html)
ZA (2) ZA201801679B (cg-RX-API-DMAC7.html)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
RS66924B1 (sr) * 2015-09-23 2025-07-31 Sangamo Therapeutics Inc Htt represori i njihova upotreba
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
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
CA3033327A1 (en) 2016-08-09 2018-02-15 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
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
AU2018355343B2 (en) * 2017-10-24 2024-12-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US11421007B2 (en) 2018-04-18 2022-08-23 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (Htt)
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
WO2020150338A1 (en) * 2019-01-15 2020-07-23 Sangamo Therapeutics, Inc. Htt repressors and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
WO2025004001A1 (en) * 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Htt repressors and uses thereof
KR102868476B1 (ko) 2025-02-20 2025-10-13 본아이에프 주식회사 냄비용 모듈형 교반기

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101754980A (zh) * 2007-05-23 2010-06-23 健泰科生物技术公司 补体相关眼疾的预防和治疗
WO2011016840A2 (en) * 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
CN104204225A (zh) * 2012-02-29 2014-12-10 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
CN104704110A (zh) * 2012-08-29 2015-06-10 桑格摩生物科学股份有限公司 用于治疗遗传性病状的方法和组合物

Family Cites Families (70)

* 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
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
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
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
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited 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
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
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
DE69940899D1 (de) 1998-05-27 2009-06-25 Genzyme Corp AAV Vektoren zur Herstellung der Medikamente zur konvektion-erhöhten Verabreichung
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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
US20100293669A2 (en) 1999-05-06 2010-11-18 Jingdong Liu Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
EP1236045B1 (en) 1999-12-06 2005-11-09 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
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
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute 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
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
EP2368982A3 (en) 2002-03-21 2011-10-12 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
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
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
CN1897977A (zh) 2003-10-20 2007-01-17 Ns基因公司 帕金森氏病的体内基因治疗
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
DK1807009T3 (en) 2004-10-05 2015-03-02 Genzyme Corp Stair shaped needle
MX2007013757A (es) 2005-05-05 2008-01-24 Univ Arizona Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas.
EP1928557B1 (en) 2005-08-23 2018-06-06 The Regents of The University of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
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
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
EP2765195A1 (en) 2006-05-25 2014-08-13 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
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
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
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
US9506120B2 (en) 2007-09-27 2016-11-29 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
WO2009151591A2 (en) 2008-06-10 2009-12-17 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
WO2010065123A1 (en) 2008-12-04 2010-06-10 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP3165234B1 (de) 2009-07-31 2019-04-03 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP2615106B1 (en) 2010-02-08 2018-04-25 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
WO2011139336A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using nucleases
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
US8945868B2 (en) 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
EP2441774B1 (en) 2010-10-15 2018-12-12 Fundació Centre de Regulació Genòmica Peptides having zinc finger domains and uses thereof
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
MX382223B (es) 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
RS66924B1 (sr) * 2015-09-23 2025-07-31 Sangamo Therapeutics Inc Htt represori i njihova upotreba

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101754980A (zh) * 2007-05-23 2010-06-23 健泰科生物技术公司 补体相关眼疾的预防和治疗
WO2011016840A2 (en) * 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
CN104204225A (zh) * 2012-02-29 2014-12-10 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
CN104704110A (zh) * 2012-08-29 2015-06-10 桑格摩生物科学股份有限公司 用于治疗遗传性病状的方法和组合物

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
"Interaction of Huntington Disease Protein with Transcriptional Activator Sp1";Shi-Hua Li et al.;《MOLECULAR AND CELLULAR BIOLOGY》;20020331;第22卷(第5期);第1277-1287页 *
"Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice";Mireia Garriga-Canut et al.;《PNAS》;20121010;第3136-3145页 *
"锌指蛋白在丝状真菌纤维素酶基因表达调控中的研究进展";杨帆等;《纤维素科学与技术》;20141231;第22卷(第4期);第64、75-81页 *

Also Published As

Publication number Publication date
HK1256102A1 (zh) 2019-09-13
FI3352776T3 (fi) 2025-07-08
US10435441B2 (en) 2019-10-08
AU2020204035A1 (en) 2020-07-09
HRP20250728T1 (hr) 2025-08-15
CL2018000749A1 (es) 2019-04-26
ZA201903325B (en) 2020-09-30
SI3352776T1 (sl) 2025-08-29
EP3352776B1 (en) 2025-03-19
BR112018005779A2 (pt) 2018-10-09
EP3352776A1 (en) 2018-08-01
EA201890493A1 (ru) 2018-09-28
KR102803519B1 (ko) 2025-05-08
WO2017053753A1 (en) 2017-03-30
ES3032775T3 (en) 2025-07-24
CN108348576A (zh) 2018-07-31
US20210369872A1 (en) 2021-12-02
PL3352776T3 (pl) 2025-09-22
IL258030B (en) 2022-08-01
ZA201801679B (en) 2019-10-30
KR20180054672A (ko) 2018-05-24
AU2016325613A1 (en) 2018-04-05
DK3352776T3 (da) 2025-06-23
JP2018532428A (ja) 2018-11-08
AU2020204035B2 (en) 2021-03-11
JP2021007405A (ja) 2021-01-28
EA037993B1 (ru) 2021-06-21
US11123443B2 (en) 2021-09-21
CA2998500A1 (en) 2017-03-30
US20200002390A1 (en) 2020-01-02
IL258030A (en) 2018-05-31
JP6853257B2 (ja) 2021-03-31
MX2018003491A (es) 2018-09-06
NZ740590A (en) 2025-05-30
EP3352776A4 (en) 2019-03-13
RS66924B1 (sr) 2025-07-31
HUE071655T2 (hu) 2025-09-28
US20170096460A1 (en) 2017-04-06
MX391052B (es) 2025-03-21
PT3352776T (pt) 2025-06-27
AU2016325613B2 (en) 2020-06-25
LT3352776T (lt) 2025-07-10

Similar Documents

Publication Publication Date Title
CN108348576B (zh) Htt阻抑物及其用途
KR102380324B1 (ko) 헌팅턴병을 치료하기 위한 방법 및 조성물
US20230270774A1 (en) Tau modulators and methods and compositions for delivery thereof
JP7542912B2 (ja) Ｈｔｔリプレッサーおよびその使用
CA2998500C (en) Htt repressors and uses thereof
HK1256102B (en) Htt repressors and uses thereof
HK1232141B (zh) 用於治療亨廷頓病的方法和組合物

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1256102

Country of ref document: HK

GR01 Patent grant
GR01 Patent grant