Jeffrey Chamberlain, PhD, on Continuing the Marathon of Muscular Dystrophy Research


The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.

“[It’s important to] recognize some of the limitations of the current gene therapies and not just saying, oh, we got one approved, let's stop there, but finding ways to increase the potency of those gene therapies and apply those same technologies to other diseases. So, the number of diseases where gene therapies are being applied now is growing significantly and hopefully, we'll have not only more and more approvals in the coming year, but continuing approvals beyond that to tweak and advance the current therapies.”

This past March, Jeffrey Chamberlain, PhD, Professor, Neurology and Medical Genetics, and Adjunct Professor, Biochemistry, and McCaw Endowed Chair of Muscular Dystrophy at University of Washington, received the MDA Legacy Award by the Muscular Dystrophy Association (MDA) at the 2024 MDA Clinical and Scientific Conference, held in Orlando, Florida. The award recognized his decades of research, going back to work he did as a graduate student, from investigating muscle specific promoters to laying the building blocks for today’s approved gene therapy for Duchenne muscular dystrophy (DMD).

CGTLive® spoke with Chamberlain during the meeting to learn more about his outlook on the field of DMD and muscular dystrophies in general, and the trajectory that research in the space might take.He stressed that while the first-of-its-kind approval was a huge step in the field, there is still much work to be done in improving therapies as well as achieving a deeper understanding of muscular dystrophies by returning to basic science.

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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