Work

RNA-based therapeutic interventions for the management of Anderson-Fabry disease

Year: 2025
Type: article
Abstract: Recently, it has been demonstrated that the modulation of the expression of alpha 1,4-galactosyltransferase (A4GALT) serves as an alternative to mitigate the accumulation of globotriaosylceramide (Gb3) in endothelial cells (ECs) and podocytes that lack galactosidase alpha (GLA).1 GLA is a gene that encodes the homodimeric glycoprotein alpha-galactosidase A (α-Gal A). This enzyme is situated within lysosomes and catalyzes the hydrolysis of terminal α-galactosyl residues from glycolipids and glycoproteins.
Institution: University of Padua
Language: English
Subfield: Physiology
Field: Medicine
Open Access status: gold
Estimated APC paid: $3,900
OpenAlex ID: W4413681407
FWCI:
Cites: 9
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Related to: 10