Onasemnogene abeparvovec is an in vivo viral-mediated gene therapy — one of only two such therapies in clinical use — which is approved for one-time intravenous administration to infants under 2 years of age with spinal muscular atrophy . Despite the small number of in vivo gene therapies in clinical use, more than a hundred are in clinical trials .
The greatest benefit from these rapidly emerging treatments is likely to come when initiated prior to disease onset, particularly when disease progression claims non-replicating cells such as neurons — as is the case for SMA. In paired papers in this issue of, Strauss et al.
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