An approach to specific therapy is perhaps the greatest need for Late Onset Tay-Sachs Disease now. Actually, a huge amount of effort has been directed toward therapy of genetic diseases. Enzyme infusion therapy has been highly effective, though very expensive, for Gaucher disease. Another approach has been gene therapy. The usual vectors chosen up to now have been viruses, especially a type of cold virus, the adenovirus.
Viruses are, of course.
Unfortunately, after a great deal of work, the result has not been successful. It isn,t entirely clear why. However, the consensus is that the problem is with the vectors. So, the focus of gene therapy has shifted towards finding better vectors, for example different viruses that might work better. Consequently, a new approach to therapy of genetic diseases is very welcome at this time.