Gene therapy holds promise for spinal muscular atrophy

US scientists have reviewed the recent developments in gene therapy as a treatment for spinal muscular atrophy. The introduction of a virus containing the SMN1 gene into mice missing this gene has proven to be very successful in the laboratory. The lessons learned from testing in mice will hopefully allow researchers to successfully test it in larger animals, paving the way for human trials.

http://www.muscular-dystrophy.org/research/news/3375_gene_therapy_holds_promise_for_spinal_muscular_atrophy