We seek to treat genetic diseases: illnesses where a single gene or several genes are either mutated or missing altogether from a person's genome. These diseases can manifest in a number of ways - illnesses as different as schizophrenia, leukemia and cystinosis can all be tied to genetic disorders. Gene therapy is the means by which we can restore faulty genes back into diseased cells or tissues to allleviate, and even cure, the diseases associated with each mutation. It is a sophisticated, highly personalized form of treatment that technology has only recently allowed humanity to harness, but we are leading the effort in this important field.

Stephanie Cherqui and Natalie's Wish: Finding a Gene Therapy for Cystinosis