The quest to cure inherited retinal diseases continues on many different fronts. It's exciting to watch the progress that the researchers are making. Everything moves slowly, one step at a time, but when taken cumulatively the pace is really quite fast. It's amazing to think how far we've come just in the past five to ten years!
Here are some articles covering recent advances:
- Tackling Inherited Blindness: This is an article from the Public Library of Science covering some of the basic approaches to curing retinal diseases. They focus on techniques used on LRAT deficient mice.
- Positive Interim Results From Phase 1b Study of Oral Synthetic Retinoid Compound in LCA: More info about the LCA "pill"—an oral medication used to treat RPE65 or LRAT.
- Second Dose of Gene Therapy for Inherited Blindness Proves Safe in Animal Studies: The gene therapy trials continue in animals and it looks like they are reacting well to being treated in the second eye.
- Oral medication for Leber's congenital amaurosis demonstrates biological signal in early study: Another article excited about the oral medication trials.
- Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of LCA: This one is just an abstract, but it reports work being done on RPGRIP1.