One year after the first human trials began in the US to test the safety of gene replacement therapy in treating a defect in the RPE65 gene which causes Leber’s Congenital Amaurosis (LCA), researchers are saying that the patients are still doing very well.
The primary purpose of this first phase of human testing is to make sure that genetic therapy is safe. They are reporting that one year after treatment, “the therapy had not provoked an immune response in the eye or in the body.”
Patients have not improved in terms of visual acuity, but they were not given significant doses of the replaced genes. However, they have reported an improvement in light perception, which is really amazing considering this trial was not intended to prove efficacy of the treatment.