Patricia Dickson: “More is better: enzyme replacement for lysosomal storage disorders”
Jonathan Cooper: “One and done? Gene therapy for lysosomal storage disorders”
There are over seventy fatal inherited diseases that kill because the lysosome, the cell’s waste disposal and recycling center, stops working properly. This is because of mutations in a gene that should make a vital part of the lysosomal machinery. In theory, this should be simple to repair either by giving back what is missing (enzyme replacement therapy) or by providing a working copy of the faulty gene that encodes the enzyme (gene therapy). We shall discuss the pluses and minuses of each approach, where we are in bringing these treatments to the clinic, and how these treatments may be commercialized.
The Institute of Clinical and Translational Sciences (ICTS) Precision Medicine team invites you to an on-going collaboration with Venture Café. These sessions will be hosted on the third Thursday of every month in the @4240 building.
For more information visit the ICTS Precision Medicine website.