May 2008

Gene Therapy: Seeing the future with new eyes

For the first time, doctors have used gene therapy to restore some vision in children with a congenital vision problem called Leber Congenital Amaurosis or “LCA” caused by a faulty gene (RPE65) 

The procedure involved the injection of a solution containing the normal gene, carried on an adenovirus vector, into the back of the retina of the affected eye.

In Britain, three patients have been treated with this experimental therapy. One has significantly improved & has been video-taped prior to the treatment & then six months later, traveling through a maze. Video HERE

The other two patients, aged 17 and 23, did not report any improvement, but did not suffer any side effects either.

Similarly, a US research team experimented on three patients, one aged 19 and two aged 26, and are reported to have improved vision as measured by standard eye tests, however, one developed a hole in the retina, thought to be due to the surgery itself.

This is a very significant milestone especially because researchers have been dealing with patients in the late stages of the disease & more hope is yet to come in those with the less-advanced form.

This is only the second time gene therapy has been proved successful in humans, after trials showed it was effective in the rare inherited disorder SCID (Severe Combined Immune Deficiency)