Regenerative medicine company jCyte and the Sue & Bill Gross Stem Cell Research Center at the University of California, Irvine report that their investigational therapy for retinitis pigmentosa (RP) has demonstrated a favorable safety and tolerability profile in an ongoing Phase I/II clinical trial. The cell-based approach taken is intended to rescue sick and dying retinal photoreceptor cells …
RP
Retinal Remodeling And Metabolic Alterations in Human AMD
Age-related macular degeneration (AMD) is a progressive retinal degeneration resulting in central visual field loss, ultimately causing debilitating blindness. While many genetic and environmental risk factors are known for AMD, we currently know less about the mechanisms mediating disease progression. The goal of this article is to illustrate cell types impacted in AMD and demonstrate the …
RetroSense Therapeutics to Present at the 8th Ocular Diseases Drug Discovery Conference
RetroSense Therapeutics is a privately-held biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). RetroSense is developing optogenetic approaches to partial vision restoration in retinitis pigmentosa and potentially dry …
Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa
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 …