Anderson–Fabry disease can be a target for gene therapy
Moataz Dowaidar
Abstract
The perspective of Anderson–Fabry disease (ADF) therapy is changing, and we should be ready to adapt to any new information. Today, ERT is still a viable therapeutic option for AFD patients. Chaperone therapy may be beneficial as a first-line treatment for adult patients with amenable mutations. In the near future, the PEGylate enzyme may be approved and added to our indications. SRT, gene therapy, and other enzyme preparations, such as moss alpha gal, are all still in the development phases.
Topics & Concepts
Enzyme replacement therapyFabry diseaseGenetic enhancementFirst line therapyAlpha-galactosidaseDiseaseMedicineGeneBioinformaticsInternal medicineBiologyGeneticsBiochemical and Molecular ResearchAmino Acid Enzymes and MetabolismCRISPR and Genetic Engineering