Written in our DNA

UF researchers’ groundbreaking work from 1980s leads to new treatment for debilitating neuromuscular disease

By Doug Bennett, Bill Levesque

An infusion of hope

Meet Londyn, the first patient in the U.S. to receive a new gene therapy for spinal muscular atrophy

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“As a pediatrician, I’m particularly grateful to see things we’ve been working on in the lab for many years impacting pediatric care. And pediatricians of the future will be able to use this therapy without having to deliver such bad news.”
Barry J. Byrne, MD, PhD
Director, UF Powell Gene Therapy Center
Professor of pediatrics
Gloved hands moving gene therapy drug The night before Londyn Wright's infusion, a drug company representative flew from Chicago to Orlando with a cooler containing four vials of Zolgensma. Another rep drove it from Orlando to Gainesville. Photo by Jesse S. Jones

A history of innovation

UF researchers were first to show AAV could be used for gene transfer in the '80s. See how it works.