Who would be eligible for gene therapy?
One of the challenges with this therapy is to determine how late in the disease process one can therapeutically intervene. Although mutations in the RPE65 gene cause severe vision loss, the structure of the retina remains intact for some indeterminate period of time. Eventually, however, retinal cells begin to die and at some point the disease is no longer treatable with gene therapy. Therefore, before a patient could enter a clinical trial it would have to be determined whether his/her retinas were still viable. Using high resolution imaging technology called Optical Coherence Tomography; NEI-supported researchers studied the structure of the retina of patients with LCA and controls without disease to determine whether it was possible to ascertain the health of the retina. Adults with LCA had evidence of thinning in some portion(s) of the retina, indicating that some cell loss had occurred. The study authors found that for the therapy to b successful in adults with LCA, it will be nec
