CN115443339A - 用于治疗亨廷顿病的方法 - Google Patents

用于治疗亨廷顿病的方法 Download PDF

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Publication number
CN115443339A
CN115443339A CN202080097236.5A CN202080097236A CN115443339A CN 115443339 A CN115443339 A CN 115443339A CN 202080097236 A CN202080097236 A CN 202080097236A CN 115443339 A CN115443339 A CN 115443339A
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China
Prior art keywords
seq
nucleic acid
sequence
isolated nucleic
raav
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Pending
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CN202080097236.5A
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English (en)
Chinese (zh)
Inventor
罗萧惠
肖啸
菲利普.穆利埃
肖斌
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.)
University of North Carolina at Chapel Hill
AskBio Inc
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University of North Carolina at Chapel Hill
Asklepios Biopharmaceutical Inc
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Publication of CN115443339A publication Critical patent/CN115443339A/zh
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    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
    • C12N9/0081—Cholesterol monooxygenase (cytochrome P 450scc)(1.14.15.6)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12Q2600/00—Oligonucleotides characterized by their use
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CN202080097236.5A 2019-12-20 2020-12-18 用于治疗亨廷顿病的方法 Pending CN115443339A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962951582P 2019-12-20 2019-12-20
US62/951,582 2019-12-20
PCT/US2020/066039 WO2021127455A1 (en) 2019-12-20 2020-12-18 Methods for treating huntington's disease

Publications (1)

Publication Number Publication Date
CN115443339A true CN115443339A (zh) 2022-12-06

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Application Number Title Priority Date Filing Date
CN202080097236.5A Pending CN115443339A (zh) 2019-12-20 2020-12-18 用于治疗亨廷顿病的方法

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US (1) US20230340470A1 (https=)
EP (1) EP4076656A4 (https=)
JP (2) JP7730327B2 (https=)
CN (1) CN115443339A (https=)
AU (1) AU2020407119A1 (https=)
CA (1) CA3163034A1 (https=)
WO (1) WO2021127455A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023211606A1 (en) * 2022-01-27 2024-07-11 AskBio Inc. Compositions for treating neurological disease

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030190746A1 (en) * 2002-04-04 2003-10-09 Xiao Xiao Gene expression control system and its use in recombinant virus packaging cell lines
WO2007089584A2 (en) * 2006-01-26 2007-08-09 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
US20080311659A1 (en) * 2005-02-11 2008-12-18 International Business Machines Corporation Ribonucleic acid interference molecules of oryza sativa
CN101365801A (zh) * 2005-10-28 2009-02-11 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
US20160296605A1 (en) * 2013-11-11 2016-10-13 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
CN106413760A (zh) * 2014-05-08 2017-02-15 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
US20180094264A1 (en) * 2016-09-22 2018-04-05 University Of Massachusetts Aav treatment of huntington's disease

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2015274457A1 (en) * 2014-06-11 2017-01-05 Circuit Therapeutics, Inc. Optogenetic therapies for movement disorders
WO2019217483A1 (en) * 2018-05-07 2019-11-14 Spark Therapeutics, Inc. Plasmid free aav vector producing cell lines
BR112022003505A2 (pt) * 2019-08-27 2022-05-24 Vertex Pharma Composições e métodos para tratamento de distúrbios associados a dna repetitivo

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030190746A1 (en) * 2002-04-04 2003-10-09 Xiao Xiao Gene expression control system and its use in recombinant virus packaging cell lines
US20080311659A1 (en) * 2005-02-11 2008-12-18 International Business Machines Corporation Ribonucleic acid interference molecules of oryza sativa
CN101365801A (zh) * 2005-10-28 2009-02-11 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
WO2007089584A2 (en) * 2006-01-26 2007-08-09 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
US20160053256A1 (en) * 2006-01-26 2016-02-25 Chdi Foundation Inc. Compositions and their uses directed to huntingtin
US20160296605A1 (en) * 2013-11-11 2016-10-13 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
CN106413760A (zh) * 2014-05-08 2017-02-15 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
US20180094264A1 (en) * 2016-09-22 2018-04-05 University Of Massachusetts Aav treatment of huntington's disease

Also Published As

Publication number Publication date
EP4076656A4 (en) 2024-07-03
EP4076656A1 (en) 2022-10-26
JP7730327B2 (ja) 2025-08-27
WO2021127455A1 (en) 2021-06-24
JP2023507741A (ja) 2023-02-27
AU2020407119A1 (en) 2022-07-14
JP2025166191A (ja) 2025-11-05
US20230340470A1 (en) 2023-10-26
CA3163034A1 (en) 2021-06-24

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