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Gene therapy for recessive RP shows preliminary proof-of-concept in pre-clinical studies

New research published in the journal Human Gene Therapy has demonstrated the rescue of a rare retinal degeneration in a recessive model of retinitis pigmentosa (RP). The research, conducted by Dr. Astra Dinculescu at the Department of Ophthalmology, University of Florida, used a viral vector to deliver a functioning copy of the MFRP (membrane-type frizzled-related protein) gene into a murine model of MFRP related RP. Results showed rescue of rod and cone photoreceptors and demonstrated successful expression of the delivered MFRP gene in the RPE (retinal pigment epithelium).