Gene therapy in Anderson-Fabry disease. State of the art and future perspectives
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 th...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2020-08-01
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| Series: | Cardiogenetics |
| Subjects: | |
| Online Access: | https://www.pagepressjournals.org/index.php/cardiogen/article/view/9075 |