Pathogenesis and Molecular Mechanisms of Anderson–Fabry Disease and Possible New Molecular Addressed Therapeutic Strategies

Pathogenesis and Molecular Mechanisms of Anderson–Fabry Disease and Possible New Molecular Addressed Therapeutic Strategies

Anderson–Fabry disease (AFD) is a rare disease with an incidenceof approximately 1:117,000 male births. Lysosomal accumulation of globotriaosylceramide (Gb3) is the element characterizing Fabry disease due to a hereditary deficiency α-galactosidase A (GLA) enzyme. The accumulation of Gb3 causes lyso...

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Bibliographic Details
Main Authors: Antonino Tuttolomondo, Irene Simonetta, Renata Riolo, Federica Todaro, Tiziana Di Chiara, Salvatore Miceli, Antonio Pinto
Format: Article
Language:English
Published: MDPI AG 2021-09-01
Series:International Journal of Molecular Sciences
Subjects:
Anderson–Fabry disease
globotriaosylceramide
endothelial dysfunction
podocyturia
valvular dysfunction
miR-1307-5p
Online Access:https://www.mdpi.com/1422-0067/22/18/10088

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