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Article

Antisense oligonucleotide mediated therapy of spinal muscular atrophy

1 Jan 2013

Abstract excerpt

Abstract Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality. SMA results from deletions or mutations of survival motor neuron 1 (SMN1), an essential gene. SMN2, a nearly identical copy, can compensate for SMN1 loss if SMN2 exon 7 skipping is prevented. Among the many cis-elements involved in the splicing regulation of SMN exon 7, intronic splicing silencer N1 (ISS-N1) has emerged as...

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PMID: 24265944
DOI: 10.2478/s13380-013-0109-2

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