UF Powell Gene Therapy Center

Tech Tuesday – Barry Byrne

In this week’s Tech Tuesday for WCJB TV20, UF Innovate’s Loren Miranda speaks with Dr. Barry Byrne, Associate Chair of Pediatrics and Director of the UF Powell Gene Therapy Center.

Atsena Therapeutics Announces Positive 12-Month Safety and Efficacy Data From Ongoing Phase I/II Clinical Trial To Treat Leber Congenital Amaurosis

Atsena Therapeutics announced positive results in their 12-month safety and efficacy data from the ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.

24-Year-Old Researches Treatment for Her Own Crippling Disease: ‘I’m in a Race Against Time’

Shandra Trantham has Friedreich's ataxia (FA), a rare genetic disease that is slowly robbing her of the ability to walk and talk, and can also affect her heart. FA is caused by an inability in the body to make frataxin, a protein that is necessary for normal cell function. To date, there is no cure and no approved treatments — but she's working on one. She's a 4th-year Ph.D. candidate in genetics and genomics at the University of Florida Powell Gene Therapy Center. Trantham works in a lab where gene therapy for FA is being fine-tuned.

Barry Byrne, MD, Ph.D.: Landscape of Awareness for Neuromuscular Disease

Barry J. Byrne, MD, Ph.D., chief medical advisor of the Muscular Dystrophy Association and associate chair of pediatrics and director at the University of Florida Powell Center for Rare Disease Research and Therapy, talked with NeurologyLive about the virtual telethon to create awareness for a number of neuromuscular diseases.