JP2022533645A - グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善 - Google Patents

グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善 Download PDF

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JP2022533645A
JP2022533645A JP2021568543A JP2021568543A JP2022533645A JP 2022533645 A JP2022533645 A JP 2022533645A JP 2021568543 A JP2021568543 A JP 2021568543A JP 2021568543 A JP2021568543 A JP 2021568543A JP 2022533645 A JP2022533645 A JP 2022533645A
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gene therapy
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glycoside hydrolase
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キャスリン クリスティーン メイヤー，
シビ リキッテ，
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リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
    • A—HUMAN NECESSITIES
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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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01018—Exo-alpha-sialidase (3.2.1.18), i.e. trans-sialidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01129—Endo-alpha-sialidase (3.2.1.129)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • 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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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Life Sciences & Earth Sciences (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
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  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
JP2021568543A 2019-05-17 2020-04-15 グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善 Pending JP2022533645A (ja)

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US201962849794P 2019-05-17 2019-05-17
US62/849,794 2019-05-17
PCT/US2020/028207 WO2020236351A1 (en) 2019-05-17 2020-04-15 Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme

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JP2022533645A true JP2022533645A (ja) 2022-07-25
JPWO2020236351A5 JPWO2020236351A5 (https=) 2023-04-24
JP2022533645A5 JP2022533645A5 (https=) 2023-04-24

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JP2021568543A Pending JP2022533645A (ja) 2019-05-17 2020-04-15 グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善
JP2021568813A Active JP7707081B2 (ja) 2019-05-17 2020-04-15 網膜細胞を標的とした最適化された遺伝子治療

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US (2) US20220233655A1 (https=)
EP (2) EP3969060A1 (https=)
JP (2) JP2022533645A (https=)
KR (1) KR20220009427A (https=)
CN (1) CN114126667A (https=)
AR (1) AR118696A1 (https=)
AU (2) AU2020278960A1 (https=)
CA (2) CA3141020A1 (https=)
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Cited By (1)

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JP2025157255A (ja) * 2019-12-25 2025-10-15 ルムス エルティーディー． 光ガイド光学素子と関連付けられた光学配置を使用して、眼からの光を方向転換することに基づく、アイトラッキングのための光学システムおよび方法

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CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
CN115323001B (zh) * 2022-07-28 2024-11-26 深圳先进技术研究院 一种靶向视网膜的基因递送系统及其应用

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AU2020278499A1 (en) 2022-01-06
KR20220009427A (ko) 2022-01-24
JP7707081B2 (ja) 2025-07-14
CA3141020A1 (en) 2020-11-26
AR118696A1 (es) 2021-10-27
WO2020236351A1 (en) 2020-11-26
EP3969060A1 (en) 2022-03-23
CA3141017A1 (en) 2020-11-26
EP3969059A1 (en) 2022-03-23
TW202110486A (zh) 2021-03-16
WO2020236352A1 (en) 2020-11-26
US20220226507A1 (en) 2022-07-21
CN114126667A (zh) 2022-03-01
US20220233655A1 (en) 2022-07-28
JP2022533983A (ja) 2022-07-27
AU2020278960A1 (en) 2021-12-23

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