Jeffrey Chamberlain, PhD, on MDA Legacy Award: From Grad Work to Gene Therapy Approval


The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed his research career with muscular dystrophies.

“It was very exciting for me to be able to receive the MDA Legacy Award this year... [in my talk] I outlined some of the things that I've done that I thought are relevant to current therapeutics... It's been very exciting for me to be able to kind of go through that arc from my early graduate career days to having a drug being approved for human use.”

The Muscular Dystrophy Association (MDA) awarded their MDA Legacy Award to Jeffrey Chamberlain, PhD, Professor, Neurology and Medical Genetics, and Adjunct Professor, Biochemistry, and McCaw Endowed Chair of Muscular Dystrophy at University of Washington, at the 2024 MDA Clinical and Scientific Conference, held March 3-6, in Orlando, Florida.

While accepting the award, Chamberlain gave an overview of his career, going all the way back to work he did as a graduate with muscle-specific promoters that would later help inform future gene therapy development and receiving his very first grant from the MDA. He discussed mouse model work that helped further understanding of Duchenne muscular dystrophy (DMD), pioneering work in delivering adeno-associated virus (AAV) vector to muscles and delivering microdystrophin with gene therapy.

CGTLive spoke with Chamberlain to hear more about his reaction to the award and his career in muscular dystrophy research. He went over his talk, how he got into gene therapy research, and shared his excitement over the recent Elevidys approval that could be seen as a culmination of his research.

Click here to view more coverage of the 2024 MDA Conference.

Muscular Dystrophy Association Announces Recipient of 2024 MDA Legacy Award for Achievement in Research, is Jeffrey Chamberlain, Ph.D., Leading Professor in Gene Therapy. News release. January 30, 2024.
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