RNA-based therapeutic interventions for the management of Anderson-Fabry disease
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.
Authors
- Stefano Cagnin (ORCID: https://orcid.org/0000-0001-7271-4625)
Institutions
- University of Padua (IT)
Publication Details
- Journal
- Molecular Therapy — Nucleic Acids
- Published
- 2025-08-26
- DOI
- https://doi.org/10.1016/j.omtn.2025.102679
- Primary Topic
- RNA regulation and disease
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ministero dell’Istruzione, dell’Università e della Ricerca
- National Institutes of Health
- Office of Research Infrastructure Programs, National Institutes of Health