New Gene Therapy for Retinitis Pigmentosa Shows Promise
FUTURE THERAPIES
RP is a rare inherited eye disease with no cure. Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. Some future treatments for retinitis pigmentosa (RP) include Stem cell therapy, gene therapy, Optogenetics, neuroprotective agents, nanobodies, N-acetylcysteine-amide (NACA).
Stem Cell Therapy | Stem cells can differentiate into various cell types, including photoreceptor cells and RPE cells, to replace lost or damaged cells.
Gene Therapy | A therapeutic gene can be injected to replace a disease-causing gene.
Optogenetics | Photosensitive proteins can be introduced into the retina to restore cone function.
Neuroprotective Agents | These include antioxidants, anti-apoptotic agents, and neurotrophic factors. They can be used in the early stages of the disease or as an adjunctive treatment.
Nanobodies | These can recognize the target rhodopsin extracellularly and lock it in a non-signaling state.
N-acetylcysteine-amide (NACA) | This molecule is designed to slow vision loss by protecting retinal cells from oxidative stress.
Other approaches under investigation include small molecules that target enzymes and receptors in cell signaling pathways involved in sight and exosomes that can deliver biologically active molecules to specific cells and tissues.