The UCSD Program for Genetic Therapies


Our Mission

We have established a program for genetic therapies that will provide a rigorous scientific and medical framework for the personalized medicine disciplines of gene therapy and genome editing. The application of powerful new genetic tools to therapy is undergoing an explosive growth following the proof of clinical efficacy of gene therapy for several otherwise intractable diseases and the emergence of genome editing as a highly promising new face for introducing therapeutic genetic changes into the human genome. Although several major genomic and gene discovery programs have recently been established at UC San Diego, none is specifically devoted to the development and application of gene-based therapies and clinically directed genome editing. UC San Diego is an ideal site for such an undertaking that will complement the advances being made in other genetics programs at UC San Diego and at our partners across the mesa, including the Center for Stem Cell Biology and Regenerative Medicine at the Sanford Burnham Prebys Medical Discovery Institute, the neonatal genetic screening program of the Rady Children’s Institute for Genomic Medicine, the basic genetics research by members of the UC San Diego Institute for Genomic Medicine (UCSD IGM), the Institute of Engineering in Medicine (UCSD IEM), and clinical research studies in the Altman Clinical and Translation Research Institute (ACTRI).
Our program fills that gap by using its resources to bring together existing UC San Diego faculty in basic and clinical research programs and in the design and implementation of clinical trials in these new fields of clinical medicine.

Theodore Friedmann, M.D., Director