There is new hope today for patients suffering from an incurable form of blindness. Researchers now explain that gene therapy might cure this type of blindness.
An unusual form of progressive blindness called choroideremia could not be cured, until now. Gene therapy might be the solution.
CBS explains how choroideremia occurs, “Caused by a mutation in the CHM gene on the X chromosome, choroideremia causes progressive blindness due to degeneration of the choroid, retinal pigment epithelium and retina. Patients with this disease can start their lives with perfect vision, but eventually start to experience problems with light sensitivity and peripheral vision as they age. The condition, which affects 1 in every 50,000 people, ultimately leads to the death of the photoreceptor cells in the retina – causing complete blindness in middle age.”
Researchers have now tested gene therapy on choroideremia patients and it has been proven to be successful. CBS sheds light on the new procedure, “Gene therapy involves injecting patients with a vital gene that is either missing or defective in their genetic code.”
The lead author of the study, from the University of Oxford, told CBS, “Gene therapy is exciting; it’s a new type of medicine. And what we’re doing is it on a very small scale, because we’re looking at a very straightforward gene to replace. It’s like looking down through a telescope at a small central island of vision. And by the time they’re in their 40s and 50s, they lose vision completely. We’re hoping to help more people with this disease, because it’s a terrible diagnosis to have. Any type of retinitis pigmentosa potentially could be helped by gene therapy. For those patients, we don’t yet know what genes to use, but the concept of this gene therapy can be applied before the onset of vision loss. So in this case, genetic modification is a good thing, not necessarily a bad thing.”