Gene Therapy For Childhood Blindness In First Clinical Trial

Science Daily- The first clinical trials for the revolutionary use of virus carrying genes to treat childhood blindness has been announced by the University College London (UCL). This kind of research is the first of its kind and will have an extraordinary impact on the future treatments of eye disease.

The trial will involve patients of all ages with Leber's congenital Amaurosis (LCA) which is an inherited type of retinal degeneration. One of the known causes of LCA is due to an abnormality in a gene called RPE65 which prevents normal function of the retina. At the present time there are no known treatments for LCA.

The new treatment will involve the injection of healthy copies of the RPE65 gene into the retina cells by using a harmless virus, also called a "vector". Restoration of these genes should restore vision. Trials on dogs with LCA produced very positive results.

I found this advancement in research particularly exciting because I personally have very horrible vision (legally blind without glasses) and have often wondered about cures for completely blind children and adults. If scientists are able to prove this method of healthy gene injection beneficial to LCA patients, it may lead to finding a way to prevent poor vision to non-LCA patients.

Source : http://www.sciencedaily.com/releases/2007/05/070501115151.htm
http://www.ucl.ac.uk/