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Article

Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAs

8 Dec 2009

Abstract excerpt

BACKGROUND: Spinal Muscular Atrophy (SMA) is an autosomal recessive disease that leads to specific loss of motor neurons. It is caused by deletions or mutations of the survival of motor neuron 1 gene (SMN1). The remaining copy of the gene, SMN2, generates only low levels of the SMN protein due to a mutation in SMN2 exon 7 that leads to exon skipping. METHODOLOGY/PRINCIPAL FINDINGS: To correct SMN2 splicing, we...

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PMID: 19997596
DOI: 10.1371/journal.pone.0008204

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