Can Gene Therapy Restore Vision in Patients with Congenital Blindness?

October 27, 2009

The use of gene therapy improved vision in all patients with Leber’s congenital amaurosis (LCA), researchers reported at the American Academy of Ophthalmology Meeting in San Francisco. These results were also simultaneously published online in The Lancet.

These results are an extension of a preliminary study published last year on three young adults, and reports successful, sustained results that showed notable improvement in children with congenital blindness. For the current human trial, the research team used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene, called RPE65 that is mutated in one form of LCA, called LCA2.

The study included five children and seven adults with LCA. The greatest improvements occurred in the children, all of whom are now able to navigate a low-light obstacle course. Although the patients did not attain normal eyesight, half of them improved enough that they may no longer be classified as legally blind.

No participants had serious adverse events from the procedure.

The researchers’ long-term goal is to administer genetic therapy as soon as a child is diagnosed in order to rescue as much of the retina as possible. The researchers also noted that further follow-up is now needed to see whether this treatment alters the natural progression of retinal degeneration.

Read the abstract/article (log in required) in The Lancet or the article here.




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