Experimental Gene Therapy Studied for Rett Syndrome

A potential therapeutic approach for Rett syndrome, an autism spectrum disorder, was published March 18 in Nature. Since a mutation in a gene called the MECP2 gene causes Rett syndrome, scientists transplanted bone marrow from healthy mice, which expressed a healthy MECP2 gene, into mice modeled to have Rett syndrome. The treatment was found to reduce disease symptoms, such as breathing and movement abnormalities, as well as to increase the lifespan of the mice.   Read More >>