Retinal degenerative diseases are major causes of untreatable blindness, and novel approaches to treatment are being sought actively. Here we explored the activation of a unique protein, sigma 1 receptor (Sig1R), in the treatment of PRC loss because of its multifaceted role in cellular survival. We used Pde6βrd10 (rd10) mice, which harbor a mutation in…
Before our brains build a visual image of our world, a chain of cells in our eyes must convert light into electrical signals that are processed in the brain. Photoreceptor cells in the retina represent the first link in this chain, and they are reactive to photons, or the fundamental particle of visible light. Retinitis pigmentosa causes these cells to…
The promising clinical results obtained for ocular gene therapy in recent years have paved the way for gene supplementation to treat recessively inherited forms of retinal degeneration. We used spliceosome-mediated RNA trans-splicing as a strategy for repairing the transcript of the rhodopsin gene, the gene most frequently mutated in autosomal dominant retinitis pigmentosa (RP). Retinitis…
Serum retinol-binding protein (RBP4) is the sole specific transport protein for retinol in the blood, but it is also an adipokine with retinol-independent, pro-inflammatory activity associated with obesity, insulin resistance, type 2 diabetes, and cardiovascular disease. Here we show that RBP4-Tg mice develop progressive retinal degeneration, characterized by photoreceptor ribbon synapse deficiency and subsequent bipolar…
Amarantus BioScience Holdings, Inc. announced the presentation of positive preclinical data for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP) at the Association for Research in Vision and Ophthalmology Annual Meeting, being held in Denver, CO. The results from preclinical studies demonstrate that MANF preserves the light-sensing function of photoreceptor cells in a genetic model of…