First pre-clinical gene therapy study to reverse Rett symptoms

(Rett Syndrome Research Trust) The concept behind gene therapy is simple: Deliver a healthy gene to compensate for one that is mutated. New research published today in the Journal of Neuroscience suggests this approach may eventually be a feasible option to treat Rett Syndrome, the most disabling of the autism spectrum disorders. Gail Mandel, Ph.D., a Howard Hughes Investigator at Oregon Health and Sciences University, led the study.
Source: EurekAlert! - Medicine and Health - Category: Global & Universal Source Type: news