Gene therapy in Anderson-Fabry disease. State of the art and future perspectives

  • Giorgio Spiniello Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Federica Verrillo Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Riccardo Ricciolino Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Dario Prozzo Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Andrea Tuccillo Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Martina Caiazza Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.
  • Marta Rubino | rubinomarta@libero.it Department of Cardiothoracic Sciences, Università degli Studi della Campania Luigi Vanvitelli, Naples, Italy.

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.

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Published
2020-08-27
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Section
Review Articles
Keywords:
Cardiomyopathies, Anderson-Fabry disease, gene therapy.
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How to Cite
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). https://doi.org/10.4081/cardiogenetics.2020.9075