In:
Cardiogenetics, MDPI AG, Vol. 10, No. 1 ( 2020-08-27), p. 9075-
Abstract:
Anderson-Fabry disease (AFD) is an X-linked lysosomal storage disorder caused by a deficiency of the lysosomal enzyme, agalactosidase A. The inadequate enzymatic activity leads to systemic storage of glycosphingolipids, mostly globotriaosylceramide, in the lysosomes. As of now, enzyme replacement therapy is the only approved treatment for AFD. However, it does not induce a complete and lasting response in several clinical contexts. Genemediated enzyme replacement is an emerging approach that could overcome these limits. The single gene nature of AFD enhances the possibility to transfect and modify a small number of cells, making them capable to affect the correction of a larger number of cells. This review summarizes the history and the state of the art of gene therapy in AFD, showing potential benefits and limits.
Type of Medium:
Online Resource
ISSN:
2035-8148
DOI:
10.4081/cardiogenetics.2020.9075
Language:
English
Publisher:
MDPI AG
Publication Date:
2020
detail.hit.zdb_id:
2628818-7