Cracking the code
UF trainees comment on UF’s role in advancing the potential of gene therapy
In June, a young patient at UF Health Shands Children’s Hospital became the first baby in the country treated with a newly approved gene therapy for a rare and debilitating neuromuscular disease since it won federal approval in May. Pioneering work done by UF researchers in the 1980s on adeno-associated virus, or AAV, made the groundbreaking treatment possible. Three former UF College of Medicine trainees comment on UF’s role in advancing the potential of gene therapy as a treatment for some of the most intractable diseases.
Arun Srivastava, PhD, the George H. Kitzman professor of genetics at UF and chief of the division of cellular & molecular therapy in the department of pediatrics, was a fellow in UF’s department of microbiology in the early 1980s, sequencing the AAV genome.
“There’s no better place than Gainesville if you’re interested in AAV gene therapy. This is the center of the AAV universe.”
Richard “Jude” Samulski, PhD ’82, is a professor of pharmacology and director of the Gene Therapy Center at the University of North Carolina. While a doctoral student at UF, he was the first researcher to clone AAV and the first to demonstrate its use as a vector.
“The stuff we did at UF launched the entire success of the community in gene therapy. It was a watershed moment. Nick’s (Muzyczka)’s lab was the one that did it for the first time and being part of that was exhilarating.”
Shannon Boye, PhD ’06, an associate professor in the department of ophthalmology at UF, leads the development of a gene therapy for Leber congenital amaurosis type 1, or LCA1, a disease where children lose their vision due to a genetic mutation that inhibits protein production in the eye’s rod and cone photoreceptors. Upon graduating from the UF College of Medicine, Boye completed a postdoctoral fellowship in the lab of UF ophthalmology professor William Hauswirth, PhD.
“The next big breakthrough in medicine is happening right now thanks to gene therapy, and UF is ground zero. We are a leader in the field and we’re getting these therapies to patients. I feel incredibly fortunate that I’ve been able to be in this field at a time when things officially started to transition from the bench to the bedside.”