Abstract
SMN1 gene is missing in SMA, however only a 5 nucleotid different, homologus, and centromeric gene called SMN2 is maintained. In RNA based therapies the main aim is to be able to obtain more SMN protein transcripts from SMN2. There is a rough inverse correlation between SMN2 copy number and clinical severity. Currently, we have two options. The first one is nusinersen, a molecule that prevents alternative splicing out of exon 7 of the SMN2 gene at the mRNA level so that more transcript is yielded.
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