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Advances in Therapeutic Development for Spinal Muscular Atrophy

1 Jun 2014

Abstract excerpt

Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. The disease originates from low levels of SMN protein due to deletion and/or mutations of SMN1 coupled with the inability of SMN2 to compensate for the loss of SMN1. While SMN1 and SMN2 are nearly identical, SMN2 predominantly generates a truncated protein (SMNΔ7) due to skipping of exon 7, the last coding exon. Several avenues for SMA...

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PMID: 25068989
DOI: 10.4155/fmc.14.63

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