CN113557243A - 用于神经变性疾病的基因疗法 - Google Patents

用于神经变性疾病的基因疗法 Download PDF

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Publication number
CN113557243A
CN113557243A CN201980086952.0A CN201980086952A CN113557243A CN 113557243 A CN113557243 A CN 113557243A CN 201980086952 A CN201980086952 A CN 201980086952A CN 113557243 A CN113557243 A CN 113557243A
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nucleic acid
isolated nucleic
vector
raav
certain embodiments
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阿萨·阿贝利奥维奇
劳拉·赫克曼
赫维·莱茵
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Privelle Therapeutics
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Privelle Therapeutics
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/31—Combination therapy
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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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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201980086952.0A 2018-11-28 2019-11-26 用于神经变性疾病的基因疗法 Pending CN113557243A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862772230P 2018-11-28 2018-11-28
US62/772,230 2018-11-28
PCT/US2019/063289 WO2020112802A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease

Publications (1)

Publication Number Publication Date
CN113557243A true CN113557243A (zh) 2021-10-26

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US (1) US20220010001A1 (https=)
EP (1) EP3887396A4 (https=)
JP (2) JP7616995B2 (https=)
KR (1) KR20210096168A (https=)
CN (1) CN113557243A (https=)
AU (1) AU2019388975A1 (https=)
BR (1) BR112021010234A2 (https=)
CA (1) CA3121211A1 (https=)
IL (1) IL283496B2 (https=)
MX (1) MX2021006253A (https=)
WO (1) WO2020112802A1 (https=)

Cited By (1)

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CN119220609A (zh) * 2024-09-27 2024-12-31 科辉智药(深圳)新药研究中心有限公司 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用

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BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP4045653A1 (en) * 2019-10-16 2022-08-24 Cornell University Gene therapy for alzheimer's disease
EP4232572A4 (en) * 2020-10-22 2024-09-25 Duke University COMPOSITIONS AND PROCEDURES RELATED TO ALZHEIMER’S DISEASE
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
EP4426833A1 (en) * 2021-11-03 2024-09-11 F. Hoffmann-La Roche AG Oligonucleotides for modulating apolipoprotein e4 expression
EP4615981A1 (en) 2022-11-11 2025-09-17 Eli Lilly and Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same

Citations (2)

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CN1092525A (zh) * 1992-10-13 1994-09-21 杜克大学 早老性痴呆的检测方法
US20150337030A1 (en) * 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors

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AU2001253233B2 (en) * 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US8435539B2 (en) * 2004-02-13 2013-05-07 Immunomedics, Inc. Delivery system for cytotoxic drugs by bispecific antibody pretargeting
CA2643048A1 (en) * 2006-02-21 2007-08-30 Oklahoma Medical Research Foundation Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
JP6469000B2 (ja) * 2012-05-18 2019-02-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイドの沈着を処置するための方法および組成物
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US20150337030A1 (en) * 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119220609A (zh) * 2024-09-27 2024-12-31 科辉智药(深圳)新药研究中心有限公司 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用
CN119220609B (zh) * 2024-09-27 2025-09-12 科辉智药(深圳)新药研究中心有限公司 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用

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US20220010001A1 (en) 2022-01-13
CA3121211A1 (en) 2020-06-04
IL283496B1 (en) 2025-05-01
IL283496B2 (en) 2025-09-01
JP7616995B2 (ja) 2025-01-17
MX2021006253A (es) 2021-09-21
JP2024177670A (ja) 2024-12-20
KR20210096168A (ko) 2021-08-04
BR112021010234A2 (pt) 2021-08-24
AU2019388975A1 (en) 2021-06-24
EP3887396A4 (en) 2022-09-07
IL283496A (en) 2021-07-29
JP2022511453A (ja) 2022-01-31
WO2020112802A1 (en) 2020-06-04
EP3887396A1 (en) 2021-10-06

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