Gene therapy for vision loss
Gene therapy for vision loss may be the cure for blindness. in December 2017, the FDA approved Luxturna. This is the first ever gene therapy to treat a genetic disease that causes blindness. In 2018, the therapy was approved.
Luxturna is designed to fix mutations in a gene that causes blindness – RPE65. One single injection in each eye is shown to be enough to improve vision for up to 3 years.
A very costly type of therapy, but Luxturna has begun many more gene therapies to fix genetic mutations that cause blindness. France is the hub where all the work is actually taking place.
Gene therapy works for blindness and provides a copy of the gene that is mutated or missing. A copy of the RPE65 gene is delivered to the patient’s eye using a viral vector. Modifications are made to eliminate its capacity for infection while allowing the translation of the DNA it carries into a functional protein.
Gene therapy is used to treat the eye. The eye is an immune privileged area of the body where the immune system cannot detect and react against the viral DNA used for delivery. The cells of the retina can keep this DNA working longer than most othyer cells in our body.
Bernard Gilly, CEO of GenSight says:
“These cells don’t renew or mutate, and therefore should conserve DNA expression all their lifetime.”
He went on to add:
“a study on neurons in monkeys showed up to eight years of expression of the protein.”
The use of gene therapy will be limited to a small number of susceptible patients with rare diseases and mutations. It will take years before gene therapy can become available to all.