JP7389744B2 - ムコ多糖症ｉｉｉａ型のための遺伝子療法 - Google Patents

ムコ多糖症ｉｉｉａ型のための遺伝子療法 Download PDF

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JP7389744B2
JP7389744B2 JP2020529562A JP2020529562A JP7389744B2 JP 7389744 B2 JP7389744 B2 JP 7389744B2 JP 2020529562 A JP2020529562 A JP 2020529562A JP 2020529562 A JP2020529562 A JP 2020529562A JP 7389744 B2 JP7389744 B2 JP 7389744B2
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aav
raav
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JP2021508241A (ja
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ウイルソン，ジェームス・エム
カッツ，ネイサン
オルドー，ジュリエット
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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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
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    • 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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    • C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
    • 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
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Virology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Plant Pathology (AREA)
  • Physical Education & Sports Medicine (AREA)
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  • 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)
  • Medicinal Preparation (AREA)
JP2020529562A 2017-11-30 2018-11-29 ムコ多糖症ｉｉｉａ型のための遺伝子療法 Active JP7389744B2 (ja)

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JP2023133281A JP2023159274A (ja) 2017-11-30 2023-08-18 ムコ多糖症ｉｉｉａ型のための遺伝子療法

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US201762593081P 2017-11-30 2017-11-30
US62/593,081 2017-11-30
PCT/US2018/063168 WO2019108857A1 (en) 2017-11-30 2018-11-29 Gene therapy for mucopolysaccharidosis iiia

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EP (1) EP3717653A4 (enExample)
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KR (1) KR102823833B1 (enExample)
AU (1) AU2018375164B2 (enExample)
BR (1) BR112020010735A2 (enExample)
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US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
US11555206B2 (en) * 2017-11-30 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIA
ES3035673T3 (en) * 2018-12-20 2025-09-08 Esteve Pharmaceuticals Sa Recombinant vectors for the long term treatment of mucchopolysacharidosis
WO2021011841A1 (en) * 2019-07-18 2021-01-21 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
AU2022388800A1 (en) * 2021-11-12 2024-06-27 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of mucopolysaccharidosis iiia
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒

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WO2019108857A1 (en) 2019-06-06
EP3717653A1 (en) 2020-10-07
AU2018375164B2 (en) 2025-05-15
KR102823833B1 (ko) 2025-06-23
IL274939A (en) 2020-07-30
US20230279430A1 (en) 2023-09-07
JP2021508241A (ja) 2021-03-04
JP2023159274A (ja) 2023-10-31
AU2018375164A1 (en) 2020-06-04
IL274939B1 (en) 2025-12-01
EP3717653A4 (en) 2021-12-01
US20200291429A1 (en) 2020-09-17
MX2020005663A (es) 2020-08-20
KR20200104307A (ko) 2020-09-03
US11555206B2 (en) 2023-01-17
IL274939B2 (en) 2026-04-01
WO2019108857A8 (en) 2020-07-16
CA3083416A1 (en) 2019-06-06
BR112020010735A2 (pt) 2020-11-10

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