Radio Boston: Tay-Sachs expert talks about gene therapy trial
Tay-Sachs disease is a devastating neurological disorder characterized by deterioration of mental and physical abilities starting at six months of age, usually resulting in death by the age of five. It is one of 40 rare, inherited metabolic disorders called lysosomal storage diseases that result from a breakdown in a cell’s ability to remove or recycle waste products due to a missing enzyme.
Dr. Sena-Esteves and his colleagues in the Tay-Sachs Gene Therapy Consortium anticipate that clinical trials might start as soon as late next year for a gene therapy treatment for Tay-Sachs.
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Related story on UMassMedNow:Tay-Sachs experts interviewed on progress of gene therapy trials