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In SMA, individuals lack a working copy of the SMN1 gene, and the ASOs are used to facilitate proper splicing of the SMN2 gene through inducing the inclusion of exon 7—ultimately rescuing the expression of the SMN protein within in vitro mammalian cell models [91] In 2016, the FDA authorized the use of Spinraza, the first drug-based therapy for SMA [93]

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