Dual Purpose Vectors for Rare Neurological Diseases

Gene therapy offers much hope for debilitating genetic disorders such as lysosomal storage diseases, which often exhibit a severe phenotype that includes neurological complications. The fact that they tend to be single gene defects and that supply of the missing enzymes through the bloodstream results in cellular uptake and translocation into lysosomes makes them amenable to gene therapy. Sandhoff and Tay-Sachs diseases, however, have additional complications that make treatment particularly challenging. A new study by Lahey et al., 1 published in this issue of Molecular Therapy, constitutes a major advance toward treatment of these devastating GM2 gangliosidoses that cause extensive damage to neurons in the brain.