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Cardiogenetics is published by MDPI from Volume 10 Issue 2 (2020). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with PAGEPress.
Review

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

Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy
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Cardiogenetics 2020, 10(1), 16-20; https://doi.org/10.4081/cardiogenetics.2020.9075
Received: 2 May 2020 / Revised: 25 June 2020 / Accepted: 20 July 2020 / Published: 27 August 2020
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
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, 16-20. 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):16-20. https://doi.org/10.4081/cardiogenetics.2020.9075

Chicago/Turabian Style

Spiniello, Giorgio; Verrillo, Federica; Ricciolino, Riccardo; Prozzo, Dario; Tuccillo, Andrea; Caiazza, Martina; Rubino, Marta. 2020. "Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives" Cardiogenetics 10, no. 1: 16-20. https://doi.org/10.4081/cardiogenetics.2020.9075

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