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 comprisin...

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Bibliographische Detailangaben
Hauptverfasser:	XIAO, Xiao, YUAN, Longhui, DU, Zengmin, JIANG, Wei, HOU, Wenxiu, CHEN, Chen, ZHENG, Hao, YANG, Caifeng, LIU, Qiang, WANG, Hui, DUN, Yue, ZHENG, Jing, ZHAO, Yang
Format:	Patent
Sprache:	chi ; eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Zusammenfassung:	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. L'invention concerne une cassette d'expression transgénique capable de traiter la mucopolysaccharidose de type IIIA, une application de celle-ci, une molécule d'acide nucléique optimisée codant pour la N-sulfoglucosamine sulfohydrolase (SGSH), un nouveau promoteur combinatoire conçu et optimisé, un système de délivrance de gènes comprenant la