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Cardiogenetics
Volume 10
Issue 1
10.4081/cardiogenetics.2020.9075
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Review

Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives

by
Giorgio Spiniello
,
Federica Verrillo
,
Riccardo Ricciolino
,
Dario Prozzo
,
Andrea Tuccillo
,
Martina Caiazza
and
Marta Rubino
*
Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy
*
Author to whom correspondence should be addressed.
Cardiogenetics 2020, 10(1), 9075; https://doi.org/10.4081/cardiogenetics.2020.9075
Submission received: 2 May 2020 / Revised: 25 June 2020 / Accepted: 20 July 2020 / Published: 27 August 2020

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.
Keywords: cardiomyopathies; Anderson-Fabry disease; gene therapy cardiomyopathies; Anderson-Fabry disease; gene therapy

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MDPI and ACS Style

Spiniello, G.; Verrillo, F.; Ricciolino, R.; Prozzo, D.; Tuccillo, A.; Caiazza, M.; Rubino, M. Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives. Cardiogenetics 2020, 10, 9075. https://doi.org/10.4081/cardiogenetics.2020.9075

AMA Style

Spiniello G, Verrillo F, Ricciolino R, Prozzo D, Tuccillo A, Caiazza M, Rubino M. Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives. Cardiogenetics. 2020; 10(1):9075. https://doi.org/10.4081/cardiogenetics.2020.9075

Chicago/Turabian Style

Spiniello, Giorgio, Federica Verrillo, Riccardo Ricciolino, Dario Prozzo, Andrea Tuccillo, Martina Caiazza, and Marta Rubino. 2020. "Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives" Cardiogenetics 10, no. 1: 9075. https://doi.org/10.4081/cardiogenetics.2020.9075

APA Style

Spiniello, G., Verrillo, F., Ricciolino, R., Prozzo, D., Tuccillo, A., Caiazza, M., & Rubino, M. (2020). Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives. Cardiogenetics, 10(1), 9075. https://doi.org/10.4081/cardiogenetics.2020.9075

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