CN112574964A - 用于治疗淀粉状蛋白沉积的方法和组合物 - Google Patents

用于治疗淀粉状蛋白沉积的方法和组合物 Download PDF

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Publication number
CN112574964A
CN112574964A CN202011133829.0A CN202011133829A CN112574964A CN 112574964 A CN112574964 A CN 112574964A CN 202011133829 A CN202011133829 A CN 202011133829A CN 112574964 A CN112574964 A CN 112574964A
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apoe
aav
protein
nucleic acid
vector
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Pending
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CN202011133829.0A
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English (en)
Chinese (zh)
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B·L·戴维森
B·T·海曼
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University of Iowa Research Foundation UIRF
General Hospital Corp
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University of Iowa Research Foundation UIRF
General Hospital Corp
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Publication of CN112574964A publication Critical patent/CN112574964A/zh
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    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • C12N15/861—Adenoviral 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202011133829.0A 2012-05-18 2013-03-14 用于治疗淀粉状蛋白沉积的方法和组合物 Pending CN112574964A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648801P 2012-05-18 2012-05-18
US61/648801 2012-05-18
CN201380038140.1A CN104540952A (zh) 2012-05-18 2013-03-14 用于治疗淀粉状蛋白沉积的方法和组合物

Related Parent Applications (1)

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CN201380038140.1A Division CN104540952A (zh) 2012-05-18 2013-03-14 用于治疗淀粉状蛋白沉积的方法和组合物

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Publication Number Publication Date
CN112574964A true CN112574964A (zh) 2021-03-30

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CN202011133829.0A Pending CN112574964A (zh) 2012-05-18 2013-03-14 用于治疗淀粉状蛋白沉积的方法和组合物
CN201380038140.1A Pending CN104540952A (zh) 2012-05-18 2013-03-14 用于治疗淀粉状蛋白沉积的方法和组合物

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Country Link
US (1) US20150183850A1 (enExample)
EP (1) EP2850195B1 (enExample)
JP (2) JP6469000B2 (enExample)
CN (2) CN112574964A (enExample)
AU (1) AU2013263346B2 (enExample)
BR (1) BR112014028666B1 (enExample)
CA (1) CA2873890C (enExample)
ES (1) ES2786078T3 (enExample)
HK (1) HK1207109A1 (enExample)
IN (1) IN2014KN02672A (enExample)
RU (1) RU2673484C2 (enExample)
WO (1) WO2013172964A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116063405A (zh) * 2022-09-27 2023-05-05 广州译码基因科技有限公司 可提高AAV病毒包装能力的衣壳蛋白突变体MutC及其应用

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CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3307391A1 (en) * 2015-06-12 2018-04-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment of alzheimer's disease
KR20180134373A (ko) * 2016-04-14 2018-12-18 타오 헬스 라이프 파마 가부시키가이샤 아밀로스페로이드(aspd)형 구조체 및 의약 조성물
CA3252099A1 (en) 2016-05-18 2025-06-05 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
AU2017306558B2 (en) * 2016-08-03 2024-09-19 University Of South Florida Reelin compositions for treatment of neurological disorders
MX2019002518A (es) * 2016-09-02 2019-07-18 Spark Therapeutics Inc Metodos y vectores para el tratamiento de trastornos del snc.
EP3309550A1 (en) * 2016-10-12 2018-04-18 sphingotec GmbH Method for the detection of apolipoprotein e4
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3121211A1 (en) * 2018-11-28 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
MX2022004524A (es) * 2019-10-16 2022-07-21 Univ Cornell Tratamiento génico para la enfermedad de alzheimer.
KR20230020946A (ko) * 2020-03-31 2023-02-13 누오-베타 파마슈티컬 테크놀로지 (상하이) 씨오., 엘티디. 중수소화 옥소페닐아르신 화합물 및 이의 용도
US20230405149A1 (en) * 2020-11-25 2023-12-21 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN120187407A (zh) * 2021-12-15 2025-06-20 健达九州(北京)生物科技有限公司 重组aav载体及其用途

Family Cites Families (10)

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US5589154A (en) * 1994-11-22 1996-12-31 Rutgers, The State University Of New Jersey Methods for the prevention or treatment of vascular hemorrhaging and Alzheimer's disease
US6436996B1 (en) * 1997-09-30 2002-08-20 Duke University Modulation of nitric oxide production
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
GB0322645D0 (en) * 2003-09-26 2003-10-29 Melacure Therapeutics Ab Use of antisecretory factor peptides
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
CN101610793B (zh) * 2006-09-14 2012-10-24 迈德詹尼克斯医疗以色列有限公司 长效药物制剂
HUE027278T2 (en) * 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
EP2561075B1 (en) * 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116063405A (zh) * 2022-09-27 2023-05-05 广州译码基因科技有限公司 可提高AAV病毒包装能力的衣壳蛋白突变体MutC及其应用

Also Published As

Publication number Publication date
EP2850195B1 (en) 2020-01-22
CN104540952A (zh) 2015-04-22
HK1207109A1 (en) 2016-01-22
JP2019031564A (ja) 2019-02-28
ES2786078T3 (es) 2020-10-08
RU2673484C2 (ru) 2018-11-27
JP6469000B2 (ja) 2019-02-13
AU2013263346B2 (en) 2018-08-23
IN2014KN02672A (enExample) 2015-05-08
JP2015520161A (ja) 2015-07-16
EP2850195A1 (en) 2015-03-25
RU2014151218A (ru) 2016-07-10
WO2013172964A1 (en) 2013-11-21
BR112014028666B1 (pt) 2021-12-07
AU2013263346A1 (en) 2014-12-04
CA2873890A1 (en) 2013-11-21
BR112014028666A2 (pt) 2017-06-27
US20150183850A1 (en) 2015-07-02
EP2850195A4 (en) 2015-12-09
CA2873890C (en) 2022-12-06

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