A beacon of hope
Barry Byrne, M.D., Ph.D., receives “See The Light” Award from the Mathew Forbes Romer Foundation…
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UF Health researchers working to develop COVID-19 vaccine using gene therapy technique
A team of University of Florida Health researchers is using an established gene therapy technique to quickly develop a COVID-19 vaccine that could be tested in humans later this year.
What’s Holding Back Gene Therapy?
Gene therapy is finally poised to revolutionize the treatment of many diseases, but the nascent field is facing challenges — notably in manufacturing — that could limit its early success, experts tell MedPage Today.
Written in our DNA
UF researchers’ groundbreaking work from 1980s leads to new treatment for debilitating neuromuscular disease…
Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy
A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at the University of Florida (UF).
UF and GoFAR announce gene therapy program for Friedreich’s ataxia
Researchers at the University of Florida have received a grant for the development of a gene therapy program aimed at treating Friedreich’s ataxia, a progressive, debilitating and life-shortening neuromuscular disorder, from the Italian patient advocacy group GoFAR.
Agilis, NCATS Partner to Advance Gene Therapy for AADC Deficiency
Agilis Biotherapeutics has been awarded a Cooperative Research and Development Agreement by National Center for Accelerating Translational Sciences (NCATS) to facilitate development of the company’s gene-therapy candidate for aromatic L-amino acid decarboxylase (AADC) deficiency.
Treatment of retinitis pigmentosa due to MERTK mutations by AAV gene vector
MERTK is an essential component of the signaling network that controls phagocytosis in retinal pigment epithelium (RPE), the loss of which results in photoreceptor degeneration. Previous proof-of-concept studies have demonstrated the efficacy of gene therapy using human MERTK (hMERTK) packaged into adeno-associated virus (AAV2) in treating RCS rats and mice…
UF researchers announce gene therapy project for Duchenne muscular dystrophy
The University of Florida’s Powell Gene Therapy Center has partnered with a biotechnology company to launch a gene therapy project aimed at developing potential treatments for Duchenne muscular dystrophy, the most common form of muscular dystrophy.
Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa
Our collaborative successful gene replacement therapy using AAV vectors expressing a variant of human RPGR-ORF15 in two canine models provided therapeutic proof of concept for translation into human treatment. The ORF15 sequence contained within this AAV vector, however, has ORF15 DNA sequence variations compared to the published sequence that are…
