Now, Nationwide researchers have shown that intravenous AAV9 gene therapy can stop spinal muscular atrophy 1 (SMA1), the most common genetic cause of death in infants. Newborns with SMA1 lack a protein needed by motor neurons in the spinal cord; the babies’ muscles weaken and eventually they cannot breathe. In November, the Nationwide team and the company AveXis reported that all but one of 12 babies who received a high dose of AAV9 carrying the gene for the missing protein can talk, eat, and sit at least briefly on their own. One girl can walk fast, and a boy is running. A new drug has achieved similar results, but it must be injected into the spine every few months.


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