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).
Gene therapy for recessive RP shows preliminary proof-of-concept in pre-clinical studies
- by swdadmin