2024 Impact Report
Showcasing the remarkable achievements of our first year. This report highlights the key milestones that have laid a strong foundation for the future of gene therapy.
Stephanie Cherqui and Natalie's Wish: Finding a Gene Therapy for Cystinosis
The story of what has driven UC San Diego's Stephanie Cherqui to develop a gene therapy for Cystinosis, a devastating inherited condition.
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 alleviate, 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.
