First pre-clinical gene therapy study to reverse Rett symptoms

The concept behind gene therapy is simple: deliver a healthy gene to compensate for one that is mutated. New research published 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. The Rett Syndrome Research Trust, with generous support from the Rett Syndrome Research Trust UK and Rett Syndrome Research & Treatment Foundation, funded this work through the MECP2 Consortium...
Source: Health News from Medical News Today - Category: Consumer Health News Tags: Autism Source Type: news