Spinal Muscular Atrophy
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YouTubeASGCT
Spinal Muscular Atrophy
Gene therapy is a fitting approach for diseases caused by a single gene mutation, like SMA. It targets the cause of disease by delivering a fully functioning copy of the gene into motor neuron cells. Let’s learn more about how it works. The subtitling of these videos was funded in part by a donation from Pfizer, Inc.
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