faculty

Jeffrey Chamberlain

jsc5@uw.edu
Professor

University of Washington

Neurology

Developmental Biology, Stem Cells & Aging

Gene Expression, Cell Cycle & Chromosome Biology

Genetics, Genomics & Evolution

Gene therapy, muscular dystrophy, CRISPR/Cas9

Faculty Contact Information

Building: S Building, South Lake Union Campus Room: S249 Box: 358055 Phone: 206-221-5363 Alt Phone: 206-616-6645

Research Summary

Our lab studies mechanisms leading to the muscular dystrophies, the function of dystrophin (DMD, among the most common inherited diseases) and FKRP (LGMD2I), and approaches to therapy. Recent work has focused on the use of AAV vectors, as we discovered that they have a remarkable ability to deliver genes bodywide via the vasculature. Significant amelioration of muscular dystrophy is achieved using systemic AAV delivery of replacement genes, or by delivery of CRISPR/Cas9 components to induce gene editing. Our vectors recently entered 2 human clinical trials for DMD.

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