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Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease

BACKGROUND: Fabry disease (FD) is a progressive multisystemic disease characterized by a lysosomal enzyme deficiency. A lack of α-galactosidase A (α-Gal A) activity results in the progressive systemic accumulation of its substrates, including globotriaosylceramide (Gb3) and globotriaosylsphingosine...

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Detalles Bibliográficos
Autores principales:	Deng, Mulan, Zhou, Hongyu, He, Shaomei, Qiu, Haoheng, Wang, Yanping, Zhao, April Yuanyi, Mu, Yunping, Li, Fanghong, Zhao, Allan Zijian
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10481556/ https://www.ncbi.nlm.nih.gov/pubmed/37670350 http://dx.doi.org/10.1186/s13023-023-02894-0

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