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 …
retinitis pigmentosa
Gene-based agents for the treatment of congenital eye diseases
Pharmacologists at LMU have developed gene-based agents for the treatment of congenital eye diseases. The first of these is now undergoing a phase-I clinical trial in color-blind patients at the University Medical Center in Tübingen. Is this approach translatable in principle to other visual disorders? Michalakis: About 200 genes have been identified which, when mutated, lead to monogenetic …
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 …
First Human Test of Optogenetics Could Restore Sight to the Blind
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 degenerate, and patients with this …
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 …
Scientists Successfully Apply Gene Therapy against Retinitis Pigmentosa
A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder. These findings are significant because they open up a new line of research to prevent nerve cell death in retinitis pigmentosa and age-related …
Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model
Gene therapy preserved vision in a study involving dogs with naturally occurring, late-stage retinitis pigmentosa, according to research funded by the National Eye Institute (NEI), part of the National Institutes of Health. The findings contribute to the groundwork needed to move gene therapy forward into clinical trials for people with the blinding eye disorder, for which there is currently no …
Amarantus Announces the Presentation of Data Showing MANF”s Positive Effect on Retinal Function at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting
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 from Eyes of Dead ‘May Give Sight to Blind’
Cells taken from the donated eyes of dead people may be able to give sight to the blind, researchers suggest. Tests in rats, reported in Stem Cells Translational Medicine, showed the human cells could restore some vision to completely blind rats. Donated corneas are already used to improve some people's sight, but the team at the Institute for Ophthalmology, at UCL, extracted a special kind of …