Dear Shahnaz, Thanks a lot for sharing this wonderful news regarding gene therapy. I am having RP. That is also considered as a hereditory disease. I hope, in future this disease will be curable with the help of gene therapy.
On 8/15/09, shahnaz <[email protected]> wrote: > timesofindia.indiatimes.com/ > Hereditary blindness curable via gene therapy > ANI 13 August 2009, 12:47pm IST > WASHINGTON: Three patients, who received gene therapy for an > inherited form of blindness, > continued having vision improvements even after a year, a new study > has revealed. > This is the first study to report one-year gene therapy safety and > efficacy results > in treating young adults with Leber Congenital Amaurosis (LCA), a > hereditary condition > that causes severe vision impairment in infants and children. > Previously, such improvement > in vision after was observed within weeks of undergoing gene therapy. > The three patients - 22, 24 and 25 years old- who received the gene > therapy, have > a specific type of LCA caused by a genetic mutation in the RPE65 gene. > The gene normally makes a critical protein in the visual cycle. > Without this RPE65 > protein, light-sensitive photoreceptor cells are starved of a > retina-specific form > of vitamin A and cannot function, blocking vision. For correcting > this genetic defect, > researchers targeted retinal regions with impaired, but intact, > photoreceptors and > injected healthy copies of the RPE65 gene under the retina. > One patient was able to read an illuminated clock for the first time, > and the new > ability was not caused by a further increase in light sensitivity, > which remained > unchanged from 1 to 12 months after the treatment. > One year after the single injection, the healthy genes continue to > make this critical > protein, increasing the retina's sensitivity to light. "We had > previously shown that > RPE65 gene therapy can completely reverse > one of the two components of this complex disease and provide > patients with increased > day and night vision within weeks. We now show that the longevity of > the visual improvements > extends to at least one year," said > Dr. Artur V. Cideciyan, Research Associate Professor of Ophthalmology > at the University > of Pennsylvania School of Medicine and lead author of the publications. > But the visual improvement was caused by a slow change in the > direction of focus > to her treated retina. The change appeared 12 months after gene > therapy, but not > before. > The findings are published in Human Gene Therapy, now online, and in > the New England > Journal of Medicine (NEJM). > More Stories from this section > High fat diet leads to short-term memory loss > Cure for multiple sclerosis now possible > Anti-psychotic drugs may fight cancer > 10 home remedies to avoid swine flu > > http://timesofindia.indiatimes.com/articleshow/msid-4889124,prtpage-1.cms > > > > Looking for local information? Find it on Yahoo! Local > http://in.local.yahoo.com/ > > > > To unsubscribe send a message to [email protected] with > the subject unsubscribe. > > To change your subscription to digest mode or make any other changes, please > visit the list home page at > http://accessindia.org.in/mailman/listinfo/accessindia_accessindia.org.in > To unsubscribe send a message to [email protected] with the subject unsubscribe. To change your subscription to digest mode or make any other changes, please visit the list home page at http://accessindia.org.in/mailman/listinfo/accessindia_accessindia.org.in
