The friendly, talkative pediatric cardiologist and molecular biologist once served as technical
adviser during the making of the Harrison Ford movie “Extraordinary Measures” and played
an extra in the film, which was based on work by Byrne and colleagues that led to an enzyme
replacement therapy for patients with Pompe disease.
In October, the FDA Cellular, Tissue and Gene Therapies Advisory Committee, with Byrne as
acting chair, recommended the approval of the gene therapy treatment for blindness, a first
for inherited genetic disorders in the U.S. The novel technique was based on groundbreaking
research by UF College of Medicine scientists, including Ken Berns, MD, PhD; Nicholas
Muzyczka, PhD; and William Hauswirth, PhD, that led to a safe method to deliver therapeutic
genes into the body.
“It is very exciting to see discoveries developed into real products that will help people who need
them the most,” Byrne said. “That is always the ultimate goal.”
FORGING RELATIONSHIPS
A key to Byrne’s success is his character. He comes
to work and makes each individual he encounters
— whether a patient, parent, staff member or
student — feel as though there’s no one else more
important to him at that moment. And as he points
to a group of framed photos in his office, it’s clear
that this loyalty to others drives his relentless pursuit
for better therapies.
“These kids are my motivation,” he said.
His impact takes on many facets. Families relocate
to Gainesville, high school students choose UF,
fellow faculty members travel across the world
(sometimes in costume) to visit kids and budding
scientists choose to study genetics as their life’s
work, with the goal of improving gene therapies.
After Gina and George Fox’s infant son, Phoenix,
was diagnosed in 2002 with Pompe disease, the
young family moved to Gainesville from the
Florida Keys, so Phoenix could receive the care
and innovative therapies offered at UF by Byrne
and his team.
“We became friends pretty much right out of the
gate because of the rareness of Phoenix’s condition,
and we could talk to him,” George Fox said of his
family’s relationship with Byrne. “To this day, if I have
any questions I can text him and he’ll answer me.
He’s so knowledgeable about the condition but he’s
also so easy to talk to.”
In 2010, sisters Maddie and Emma Crowley, who
each had recently been diagnosed with Pompe,
traveled with their parents from Philadelphia to
Gainesville to find answers to what they only knew
as an incurable, degenerative disease that would
affect the rest of their lives. In Byrne, the two young
girls met someone who would become a lifesaver
and a life changer.
“Dr. Byrne is incredibly real and thoughtful,”
Crowley said. “He will do all these tests with us as
patients and then will go out to dinner with us or
just be crazy. One time, on a tour of UF when we
GO
GREATER
were younger, he snuck us onto Florida Field and ran
across the field with Maddie."
Today, Maddie is a UF student and Emma works as
a research coordinator in Byrne’s lab.
Barbara Smith, PT, PhD, an assistant professor
in the colleges of Medicine and Public Health and
Health Professions and a colleague of Byrne’s at
the Powell Center for Rare Disease Research and
Therapy, often travels with him and no longer thinks
twice about donning a Halloween costume and
arriving at a patient’s home as “Star Wars” villain
Darth Vader, while Byrne dresses up as a character
from the film “Avatar.”
Angela McCall, PhD ’17, a recent graduate of
the UF College of Medicine biomedical sciences
graduate program, attributes her success to Byrne,
her mentor, and his ability to not only teach students
about genetics and research, but also how to be
well-rounded individuals who can offer more than
the facts.
McCall and other recent doctoral program
graduates mentored by Byrne have gone on to
continue their research at institutions like Duke
University, the Dana-Farber Cancer Institute and St.
Jude Children’s Research Hospital, with the goal of
studying and using gene therapies to treat diseases.
“I credit a lot of what I’m doing now to him,”
McCall said. “I’m able to do these things because of
what he taught and required of his students.”
The FDA approval of a gene therapy treatment in
December for people with an inherited eye disease
was a significant move, says Byrne, that provides a
road map for making other gene therapies available
for even more diseases. Approval is “critical to the
sustainability” of this treatment method, he said.
“If we have enough evidence that this idea of
a replacement gene is safe,” Byrne said, “then
we can show it will improve lives and people will
adopt that idea.
“I think we’re at the tipping point for that.”
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