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Latest RPE65 Stories

Researcher Reveals Novel Treatment For A Form Of Childhood Blindness
2014-07-14 03:18:37

McGill University Health Centre 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). Until now, this inherited retinal disease that causes visual impairment ranging from reduced vision to complete blindness, has remained untreatable. The study is published today in the scientific...

2014-05-27 16:25:11

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/ -- Spark Therapeutics, a late-stage company developing gene-based medicines for a wide range of rare, debilitating diseases, today announced the successful completion of a $72.8 million Series B financing led by Sofinnova Ventures. Joining Sofinnova as new investors in the company are Brookside...

2014-03-25 00:23:24

PHILADELPHIA, March 25, 2014 /PRNewswire/ -- Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the...

2014-01-14 12:26:51

Spark CEO provides corporate updates at the J.P. Morgan Conference SAN FRANCISCO, Jan. 14, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company, has reached its patient recruitment goal for its most advanced clinical program, a Phase 3 study for inherited retinal dystrophies caused by mutations in the RPE65 gene. Spark's CEO, Jeffrey D. Marrazzo, is presenting this and other corporate updates today, at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco....

2013-02-13 11:44:06

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. This protein may provide a new therapeutic target for both an inherited retinal degenerative disease and age-related macular degeneration. The paper is published in the February 13, 2013 issue of the Journal of Neuroscience....

2009-08-13 09:47:18

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. One patient also noticed a visual improvement that helped her perform daily tasks, which scientists describe in an Aug. 13 letter to the editor in the New England Journal of Medicine.These findings have emerged from a phase I clinical trial supported by the National Eye Institute (NEI) at the...

2005-08-11 19:30:00

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. The study, "Rpe65 is the Retinoid Isomerase in Bovine Retinal Pigment Epithelium," is published in the Aug. 12 issue of Cell magazine. "We were amazed when we...


Word of the Day
endocarp
  • The hard inner (usually woody) layer of the pericarp of some fruits (as peaches or plums or cherries or olives) that contains the seed.
This word comes from the Greek 'endon,' in, within, plus the Greek 'kardia,' heart.
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