TRANSGENIC EXPRESSION CASSETTE FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS TYPE IIIA
Provided are a transgenic expression cassette capable of treatment of mucopolysaccharidosis type IIIA, an application thereof, an optimized nucleic acid molecule encoding N-sulfoglucosamine sulfohydrolase (SGSH), a designed and optimized novel combinatorial promoter, a gene delivery system comprising the transgenic expression cassette, and a drug. The transgenic expression cassette, the gene delivery system, and the drug can effectively restore SGSH activity in patients with mucopolysaccharidosis, reduce accumulation of glycosaminoglycans, and have a good treatment effect on peripheral body diseases and central nervous system diseases of mucopolysaccharidosis type IIIA..
| Medienart: |
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Patent| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Europäisches Patentamt - (2023) vom: 07. Dez. Zur Gesamtaufnahme - year:2023 |
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| Sprache: |
Englisch |
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| Beteiligte Personen: |
XIAO XIAO [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2023-12-07, Last update posted on www.tib.eu: 2024-01-03, Last updated: 2024-01-12 |
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| Patentnummer: |
WO2023231778 |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
EPA018861636 |
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| 520 | |a Provided are a transgenic expression cassette capable of treatment of mucopolysaccharidosis type IIIA, an application thereof, an optimized nucleic acid molecule encoding N-sulfoglucosamine sulfohydrolase (SGSH), a designed and optimized novel combinatorial promoter, a gene delivery system comprising the transgenic expression cassette, and a drug. The transgenic expression cassette, the gene delivery system, and the drug can effectively restore SGSH activity in patients with mucopolysaccharidosis, reduce accumulation of glycosaminoglycans, and have a good treatment effect on peripheral body diseases and central nervous system diseases of mucopolysaccharidosis type IIIA. | ||
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