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Stephanie Salabarria

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.

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…