View Full Version : Genetic Testing and Gene Therapy for Leber's Congenital Amaurosis


Andrew_Doan
06-22-2005, 05:12 PM
http://webeye.ophth.uiowa.edu/eyeforum/Genetic-Testing-for-Lebers-Congenital-Amaurosis.htm

by Edwin Stone, MD, PhD

Presented at the New England Ophthalmological Society

thalia44
10-26-2009, 09:31 AM
There was more news about this from UPenn/Naples and Dr. Katherine A. High, Dr. Jean Bennett, and Dr. Albert M. Maguire.

LATimes: Gene therapy transforms eyesight of 12 born with rare defect (http://www.latimes.com/news/nationworld/nation/la-sci-gene-therapy25-2009oct25,0,2334183.story)

The original article is in the Lancet (available online if you have access)

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial (http://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2809%2961836-5/abstract)

At the end of the article online, there are videos of patients walking through an obstacle course before and after treatment... it's pretty incredible!