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

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 study, conducted by Dr. Astra Dinculescu and colleagues 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 indicated the rescue of rod and cone photoreceptors and showed successful expression of the delivered MFRP gene in the RPE (retinal pigment epithelium). Further efficacy, safety and dose response analysis, in addition to developing a potential intravitreal route of delivery, are proposed prior to any assessment of suitability for human clinical studies.