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Genetic Diseases and Gene Therapy
The overall focus of this laboratory is therapy for inherited metabolic diseases, and a recurring theme is the use of mouse genetics and mouse models of human disease. In particular gene therapy approaches are being explored. In the past two lysosomal storage diseases, MPS I and MPS VII, were treated with remarkable success in mouse models. Adeno-associated viral vectors were used to deliver functional gene substitutes for the defective genes that cause these diseases. Currently, research efforts center primarily on Smith-Lemli-Opitz syndrome (SLOS), which is caused by an inherited deficiency in cholesterol synthesis. Because cholesterol has multiple, essential roles in normal physiology and development, SLOS is characterized by a wide variety of problems including dysmorphias (e.g., cleft lip), growth delay, and mental retardation. There is no known cure for SLOS. In the Watson lab, mouse models of SLOS are being employed to explore the possibility of gene therapy for this disease, and results so far are encouraging.


If you are interested in making a donation to help further our research, please select UCSF Benioff Children's Hospital Foundation and click on the 'Donate' button in the middle of the page. Select 'other' for the 'I want my funds to support' and type Gordon Watson, research project: Gene Therapy for Inherited Diseases, cost center: 12.7871.

Alternatively, you may send a check to the UCSF Benioff Children's Hospitals Foundation at 2201 Broadway, Suite 600, Oakland, CA 94612. Clearly mark your check or cover letter so that your gift will be directed to support this work. To make a gift of securities in support of research, please telephone the Foundation at (510) 428-3814.




Revised: Tuesday, April 7, 2015 5:19 PM


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