CN104540952A - 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
CN104540952A
CN104540952A CN201380038140.1A CN201380038140A CN104540952A CN 104540952 A CN104540952 A CN 104540952A CN 201380038140 A CN201380038140 A CN 201380038140A CN 104540952 A CN104540952 A CN 104540952A
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CN
China
Prior art keywords
apoe
aav
nucleic acid
ependymocyte
mammiferous
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Pending
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CN201380038140.1A
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English (en)
Chinese (zh)
Inventor
B.L.戴维森
B.T.海曼
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 Iowa Research Foundation UIRF
General Hospital Corp
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University of Iowa Research Foundation UIRF
General Hospital Corp
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Application filed by University of Iowa Research Foundation UIRF, General Hospital Corp filed Critical University of Iowa Research Foundation UIRF
Priority to CN202011133829.0A priority Critical patent/CN112574964A/zh
Publication of CN104540952A publication Critical patent/CN104540952A/zh
Pending legal-status Critical Current

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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
    • 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
    • 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
    • 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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CN201380038140.1A 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 CN104540952A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202011133829.0A 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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648801P 2012-05-18 2012-05-18
US61/648801 2012-05-18
PCT/US2013/031725 WO2013172964A1 (en) 2012-05-18 2013-03-14 Methods and compositions for treating amyloid deposits

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202011133829.0A Division 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

Publications (1)

Publication Number Publication Date
CN104540952A true CN104540952A (zh) 2015-04-22

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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
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

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Country Link
US (1) US20150183850A1 (enExample)
EP (1) EP2850195B1 (enExample)
JP (2) JP6469000B2 (enExample)
CN (2) CN104540952A (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 (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109072274A (zh) * 2016-04-14 2018-12-21 道健康生活医药株式会社 淀粉样蛋白球体（aspd）样结构体及医药组合物
CN109891240A (zh) * 2016-10-12 2019-06-14 斯弗因高泰克有限公司 载脂蛋白e4的检测方法
CN110121356A (zh) * 2016-09-02 2019-08-13 星火治疗股份有限公司 治疗cns疾病的方法和载体
CN115515974A (zh) * 2020-03-31 2022-12-23 挪贝肽医药科技(上海)有限公司 Pi4k抑制剂在细胞内蛋白错误折叠相关疾病和溶酶体贮积病中的应用
WO2023108507A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Recombinant aav vectors and use thereof

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KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016196507A1 (en) 2015-05-29 2016-12-08 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
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
WO2018027037A2 (en) * 2016-08-03 2018-02-08 University Of South Florida Reelin compositions for treatment of neurological disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
CN114761569A (zh) * 2019-10-16 2022-07-15 康奈尔大学 阿尔茨海默氏病的基因疗法
CA3203006A1 (en) * 2020-11-25 2022-06-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN116063405B (zh) * 2022-09-27 2025-08-29 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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CN101754682A (zh) * 2007-05-16 2010-06-23 布里格姆妇女医院 突触核蛋白病的治疗

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CA2591544A1 (en) * 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
MX2007013734A (es) * 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
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WO1999016464A1 (en) * 1997-09-30 1999-04-08 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
CN101754682A (zh) * 2007-05-16 2010-06-23 布里格姆妇女医院 突触核蛋白病的治疗

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FENG XUAN ET AL: "Adeno-associated viral vector-mediated ApoE expression in Alzheimer"s disease mice: low CNS immune response , long-term expression , and astrocyte specificity", 《FRONTIERS IN BIOSCIENCE》 *
JEAN-COSME DODART ET AL: "Gene delivery of human apolipoprotein E alters brain Aβburden in a mouse model of Alzheimer’s disease", 《PNAS》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109072274A (zh) * 2016-04-14 2018-12-21 道健康生活医药株式会社 淀粉样蛋白球体（aspd）样结构体及医药组合物
CN110121356A (zh) * 2016-09-02 2019-08-13 星火治疗股份有限公司 治疗cns疾病的方法和载体
CN109891240A (zh) * 2016-10-12 2019-06-14 斯弗因高泰克有限公司 载脂蛋白e4的检测方法
CN115515974A (zh) * 2020-03-31 2022-12-23 挪贝肽医药科技(上海)有限公司 Pi4k抑制剂在细胞内蛋白错误折叠相关疾病和溶酶体贮积病中的应用
WO2023108507A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Recombinant aav vectors and use thereof

Also Published As

Publication number Publication date
JP2015520161A (ja) 2015-07-16
EP2850195A4 (en) 2015-12-09
CA2873890A1 (en) 2013-11-21
IN2014KN02672A (enExample) 2015-05-08
ES2786078T3 (es) 2020-10-08
JP6469000B2 (ja) 2019-02-13
RU2014151218A (ru) 2016-07-10
JP2019031564A (ja) 2019-02-28
WO2013172964A1 (en) 2013-11-21
AU2013263346A1 (en) 2014-12-04
EP2850195A1 (en) 2015-03-25
CA2873890C (en) 2022-12-06
BR112014028666A2 (pt) 2017-06-27
HK1207109A1 (en) 2016-01-22
CN112574964A (zh) 2021-03-30
EP2850195B1 (en) 2020-01-22
US20150183850A1 (en) 2015-07-02
AU2013263346B2 (en) 2018-08-23
BR112014028666B1 (pt) 2021-12-07
RU2673484C2 (ru) 2018-11-27

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Application publication date: 20150422

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