Latest RPE65 Stories
Company on track to report Phase 3 data in second half of 2015 PHILADELPHIA, Nov.
An international research project, led by the Research Institute of the McGill University Health Centre (RI-MUHC) in Montreal, reports that a new oral medication is showing significant progress in restoring vision to patients with Leber congenital amaurosis (LCA).
Gene therapy leader to advance its deep pipeline of rare disease programs including its lead Phase 3 product candidate for RPE65-related blindness PHILADELPHIA, May 27, 2014 /PRNewswire/
PHILADELPHIA, March 25, 2014 /PRNewswire/ -- Spark Therapeutics and Genable
Spark CEO provides corporate updates at the J.P. Morgan Conference SAN FRANCISCO, Jan.
Research led by Minghao Jin, PhD, Assistant Professor of Ophthalmology and Neuroscience at the LSU Health Sciences Center New Orleans Neuroscience Center of Excellence, has found a protein that protects retinal photoreceptor cells from degeneration caused by light damage.
Three young adults who received gene therapy for a blinding eye condition remained healthy and maintained previous visual gains one year later, according to an August online report in Human Gene Therapy.
In findings that could lead to curing some forms of congenital blindness through gene therapy, researchers at UCLA have discovered that RPE65, a gene missing in infants born with the blinding disease Leber congenital amaurosis, is also a key enzyme in the visual cycle. The identity of this enzyme has long been a mystery to scientists.