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

Abstract 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 globotriaosylsphi...

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Hlavní autoři:	Mulan Deng, Hongyu Zhou, Shaomei He, Haoheng Qiu, Yanping Wang, April Yuanyi Zhao, Yunping Mu, Fanghong Li, Allan Zijian Zhao
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2023-09-01
Edice:	Orphanet Journal of Rare Diseases
Témata:	Fabry disease (FD) Adeno-associated viral 2/8 (AAV2/8) α-galactosidase A (α-Gal A) Globotriaosylceramide (Gb3) Globotriaosylsphingosine (Lyso-Gb3) Lysosomal storage diseases (LSDs)
On-line přístup:	https://doi.org/10.1186/s13023-023-02894-0
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