Professional Overview
Administrative Appointments
- Chair, School of Medicine Appointments and Promotions Committee (2008 - 2010)
Honors and Awards
- Searle Scholar Award, Searle Family Foundation (1983 - 1986)
- Graduate Fellowship, National Science Foundation (1979)
Professional Education
| B.A., M.A.: | Oxford University, Zoology |
| Ph.D.: | Harvard University, Biochemistry & Molecular Biology |
| Postdoc.: | University of Geneva, Biologie Moleculaire |
Graduate & Fellowship Program Affiliations
Community and International Work
- Member, Board of Directors, American Society of Gene and Cell Therapy, International
- Advisory Committee, United States Food and Drug Administration, Bethesda, Maryland
Internet Links
Industry Relationships
Stanford is committed to ethical and transparent interactions with our industrial and other commercial partners. It is our policy to disclose payments (exclusive of travel support) from, and/or equity in, companies or other commercial entities to Stanford faculty of $5,000 or more in total value, as well as any equity in a privately held company, when the faculty member also has institutional responsibilities related to his or her interactions with the company. View Full Information
Scientific Focus
Current Research Interests
Our research is focused on development of novel strategies for gene and cell therapy, using regenerative medicine approaches. We would like to improve the clinical condition of patients suffering from genetic disorders like Duchenne muscular dystrophy and limb girdle muscular dystrophy type 2B and degenerative diseases like Parkinson's disease.
To develop a therapy for muscular dystrophy, we are using induced pluripotent stem cells (iPSC) derived from patients, using non-viral reprogramming methods. We carry out precise genome engineering to add therapeutic sequences to the iPSC genome, using homologous recombination and phage integrases to mediate sequence-specific genomic integration. The corrected cells are differentiated into muscle precursors in vitro, then transplanted back to the patient, where they can engraft and produce healthy muscle fibers.
We are currently testing these approaches in mouse models of muscular dystrophy. If successful, these types of therapies will provide new options for patients suffering from genetic diseases. They may also provide new possibilities for treatment of other common diseases and conditions, including normal aging.
Publications
- Efficient reversal of phiC31 integrase recombination in mammalian cells. Biotechnol J. 2012; (11): 1332-6
- Long-term expression of human coagulation factor VIII in a tolerant mouse model using the φC31 integrase system. Hum Gene Ther. 2012; (4): 390-8
- Safe genetic modification of cardiac stem cells using a site-specific integration technique. Circulation. 2012; (11 Suppl 1): S20-8
- Long-term phenotypic correction in factor IX knockout mice by using ΦC31 integrase-mediated gene therapy. Gene Ther. 2011; (8): 842-8
- Site-specific recombinase strategy to create induced pluripotent stem cells efficiently with plasmid DNA. Stem Cells. 2011; (11): 1696-704
- The therapeutic potential of ΦC31 integrase as a gene therapy system. Expert Opin Biol Ther. 2011; (10): 1287-96
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