Alumni News
Cracking the code
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. Please see page 16 for the full story.
34 | F LO R I DA P HYS I C IAN
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.”
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.”
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.”
PHOTO BY JESSE S. JONES
PHOTO BY TRICIA COYNE
PHOTO BY MINDY C. MILLER