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Gene Therapy for Sanfilippo: Where We Are Now
Gene therapy is a promising approach for a number of rare genetic diseases as it has the potential to treat the underlying cause. Here is more information about gene therapy, its challenges, and its current status as a treatment for Sanfilippo syndrome.
Sanfilippo syndrome is caused by mutations in genes that encode for enzymes which break down long chains of sugar molecules. The goal of gene therapy for Sanfilippo is to deliver correct copies of the faulty gene to patients so that their cells can produce functional enzymes to breakdown and prevent the accumulation of these sugar molecules.
The first is that each of the four subtypes of Sanfilippo syndrome is due to a mutation in a different gene, so each subtype requires a separate target for gene therapy. In other words, one gene therapy will not work for all Sanfilippo patients.
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