JP2023524010A - 高効率遺伝子送達系 - Google Patents

高効率遺伝子送達系 Download PDF

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Publication number
JP2023524010A
JP2023524010A JP2022565920A JP2022565920A JP2023524010A JP 2023524010 A JP2023524010 A JP 2023524010A JP 2022565920 A JP2022565920 A JP 2022565920A JP 2022565920 A JP2022565920 A JP 2022565920A JP 2023524010 A JP2023524010 A JP 2023524010A
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Japan
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mirna
delivery system
viral vector
viral
apeinfo
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JP2022565920A
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Japanese (ja)
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JP2023524010A5 (https=
Inventor
マイケル フローリア，
エイミー ジェイ． ウェイジャーズ，
ルーク ヴァンデンベルグ，
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Harvard University
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Harvard University
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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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022565920A 2020-04-28 2021-04-28 高効率遺伝子送達系 Pending JP2023524010A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063016968P 2020-04-28 2020-04-28
US63/016,968 2020-04-28
PCT/US2021/029757 WO2021222476A2 (en) 2020-04-28 2021-04-28 High efficiency gene delivery system

Publications (2)

Publication Number Publication Date
JP2023524010A true JP2023524010A (ja) 2023-06-08
JP2023524010A5 JP2023524010A5 (https=) 2024-05-10

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ID=78374241

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022565920A Pending JP2023524010A (ja) 2020-04-28 2021-04-28 高効率遺伝子送達系

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US (2) US20220193262A1 (https=)
EP (1) EP4142758A4 (https=)
JP (1) JP2023524010A (https=)
WO (1) WO2021222476A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240002459A1 (en) * 2016-07-14 2024-01-04 Emory University Granulin Compositions and Uses Related Thereto
CN114085872A (zh) * 2021-11-10 2022-02-25 中国科学院深圳先进技术研究院 一种表达tva的小鼠模型的构建方法和应用
CN115029346B (zh) * 2022-04-22 2024-01-02 复旦大学附属眼耳鼻喉科医院 用于靶向敲降Htra2转录本的sgRNA、CRISPR/CasRx系统及应用
WO2024057339A1 (en) * 2022-09-16 2024-03-21 National Institute Of Immunology Itch mutants for the treatment of memory and learning defects
WO2025094056A1 (en) * 2023-10-31 2025-05-08 Children's Medical Research Institute Gene therapy constructs and methods of use therefor

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110185440A1 (en) * 2006-10-03 2011-07-28 National Yang-Ming University CISD2-Knockout Mice and Uses Thereof
JP2019519221A (ja) * 2016-05-20 2019-07-11 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3318635A1 (en) * 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
TWI614031B (zh) * 2011-11-03 2018-02-11 國立陽明大學 防止皮膚老化相關症狀的醫藥組合物及其使用方法
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
WO2016077706A1 (en) * 2014-11-13 2016-05-19 Washington University Treatment for wolfram syndrome and other endoplasmic reticulum stress disorders
US20160362692A1 (en) * 2015-06-15 2016-12-15 Isis Innovation Limited Treatment of retinitis pigmentosa
MA51915A (fr) * 2018-02-22 2020-12-30 Generation Bio Co Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110185440A1 (en) * 2006-10-03 2011-07-28 National Yang-Ming University CISD2-Knockout Mice and Uses Thereof
JP2019519221A (ja) * 2016-05-20 2019-07-11 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, vol. 1201, JPN6025024873, 2010, pages 58 - 64, ISSN: 0005773356 *

Also Published As

Publication number Publication date
EP4142758A4 (en) 2025-02-26
WO2021222476A2 (en) 2021-11-04
EP4142758A2 (en) 2023-03-08
US20250325701A1 (en) 2025-10-23
WO2021222476A3 (en) 2021-12-09
US20220193262A1 (en) 2022-06-23

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