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A beacon of hope

Barry Byrne, M.D., Ph.D., receives “See The Light” Award from the Mathew Forbes Romer Foundation…

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…

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…