She is 14 on May 1st! :-)
The Telethon Institute of Genetics and Medicine in Naples explains the hope for a cure of Leber's congenital amaurosis, a genetic disease affecting the eyes that leads to blindness a few months after birth, thanks to gene therapy. The Vision of Children is not responsible for the content of this video, but we think it is a great step in the right direction towards the cure of genetic eye diseases.
In 2009, Dr. Hauswirth completed a successful clinical trial where children with the retinal eye disorder Leber Congenital Amaurosis (LCA), regained eyesight. Although the first human trials in gene therapy were to treat LCA, leading research institutions hope that a similar process can also be used to treat other vision disorders. VOC is currently providing Dr. Hauswirth with a research grant to use his findings with LCA to treat patients with BCM using retinal gene therapy.
Through generous donor support, The Vision of Children has funded the research of Dr. William Hauswirth of the University of Florida. Hauswirth and his team have conducted extensive research in the field of gene therapy in an effort to cure various retinal eye disorders. For the past three years, they have been working towards a human clinical trial to cure Blue Cone Monochromacy, a rare form of color blindness. This video highlights some of the achievements made by his research team.
Check out the research being done at the Macula Vision Research Foundation in Israel. The Vision of Children Foundation did not fund this specific gene therapy trial, however we think it is a great demonstration of the direction in which we hope to move towards. We are moving forward together!