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Center for Gene Therapy

Visualizing Gene Therapy for SMA
 Visualizing Gene Therapy for SMA 150 150 Abbie Miller
By: Abbie Miller

Spinal muscular atrophy (SMA), a degenerative neuromuscular disease, is the most common genetic cause of death for infants. Virtually all children affected with SMA type 1 die by 2 years of age. In 2014, Jerry Mendell, MD, director of the Center for Gene Therapy in the Research Institute at Nationwide Children’s Hospital, began a phase 1 gene transfer clinical…

Gene Therapy’s Road to Redemption
 Gene Therapy’s Road to Redemption 150 150 Kelli Whitlock Burton
By: Kelli Whitlock Burton

Fifteen years ago, gene therapy suffered a highly visible fatality, leaving the field in shambles. Now, one team’s efforts at gene therapy for muscular dystrophy suggest the field may finally be on track to deliver on its initial promise. During the first few weeks of September in 1999, a 36-year-old air traffic controller from South…

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