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Bruce Conklin, MD, and his team at Gladstone Institutes are tackling Charcot-Marie-Tooth (CMT) disease, the most common inherited neurological disorders—for which there is currently no cure.
Using cells collected from patients with CMT, the scientists are exploring ways to use gene editing as a therapeutic tool to treat the disease.
In this video, Bruce Conklin and Bria Macklin describe their work and explain how it could help patients like Jeremy, an energetic 5-year-old who donated his cells to Gladstone for this research.
Content note: The narration in the first 6 seconds of this video was enhanced using an AI-generated voice.
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