2013•BioDrugsRequires access

Gene Therapy for Fabry Disease: A Review of the Literature

Aritz Pérez Ruiz de Garibay, Maria Ángelés Solinís, Alicia Rodríguez‐Gascón

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Key concepts: Globotriaosylceramide, Fabry disease, Enzyme replacement therapy, Genetic enhancement, Alpha-galactosidase, Disease, Lysosomal storage disease, Transfection

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Gene Therapy for Fabry Disease: A Review of the Literature — Research Paper | ScholarLens