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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Prior art keywords
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)
Inventor
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)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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)
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

Publications (1)

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

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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 (https=)
EP (1) EP2850195B1 (https=)
JP (2) JP6469000B2 (https=)
CN (2) CN104540952A (https=)
AU (1) AU2013263346B2 (https=)
BR (1) BR112014028666B1 (https=)
CA (1) CA2873890C (https=)
ES (1) ES2786078T3 (https=)
HK (1) HK1207109A1 (https=)
IN (1) IN2014KN02672A (https=)
RU (1) RU2673484C2 (https=)
WO (1) WO2013172964A1 (https=)

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

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230145206A (ko) 2014-11-14 2023-10-17 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
US20180161395A1 (en) * 2015-06-12 2018-06-14 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and pharmaceutical composition for the treatment of alzheimer's disease
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
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
AU2017306558B2 (en) * 2016-08-03 2024-09-19 University Of South Florida Reelin compositions for treatment of neurological disorders
JP7610923B2 (ja) * 2016-09-02 2025-01-09 スパーク セラピューティクス インコーポレイテッド Ｃｎｓ障害を治療するための方法及びベクター
EP3309550A1 (en) * 2016-10-12 2018-04-18 sphingotec GmbH Method for the detection of apolipoprotein e4
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
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
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
JP2023500793A (ja) * 2019-10-16 2023-01-11 コーネル・ユニバーシティー アルツハイマー病に対する遺伝子治療方法
EP4065600A1 (en) 2019-11-25 2022-10-05 Cornell University Apoe gene therapy
BR112022019864A2 (pt) * 2020-03-31 2022-11-16 Nuo Beta Pharmaceutical Tech Shanghai Co Ltd Composto de oxofenilarsina deuterada e uso do mesmo
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
WO2023108507A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Recombinant aav vectors and use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP5136766B2 (ja) * 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
CA2606490C (en) * 2005-05-02 2018-02-06 Genzyme Corporation Gene therapy for spinal cord disorders
CN102886052B (zh) * 2006-09-14 2014-07-30 迈德詹尼克斯医疗以色列有限公司 长效药物制剂
EP2154969B1 (en) * 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
EP3444346B1 (en) * 2010-04-23 2022-07-27 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
CA2873890C (en) 2022-12-06
RU2673484C2 (ru) 2018-11-27
CN104540952A (zh) 2015-04-22
EP2850195B1 (en) 2020-01-22
US20150183850A1 (en) 2015-07-02
HK1207109A1 (en) 2016-01-22
RU2014151218A (ru) 2016-07-10
AU2013263346A1 (en) 2014-12-04
JP6469000B2 (ja) 2019-02-13
EP2850195A4 (en) 2015-12-09
BR112014028666A2 (pt) 2017-06-27
IN2014KN02672A (https=) 2015-05-08
AU2013263346B2 (en) 2018-08-23
BR112014028666B1 (pt) 2021-12-07
JP2019031564A (ja) 2019-02-28
EP2850195A1 (en) 2015-03-25
ES2786078T3 (es) 2020-10-08
WO2013172964A1 (en) 2013-11-21
CA2873890A1 (en) 2013-11-21
JP2015520161A (ja) 2015-07-16

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