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Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector

1 Apr 2006

Abstract excerpt

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that is the leading genetic cause of infant mortality. SMA is caused by the loss of survival motor neuron-1 (SMN1). In humans, a nearly identical copy gene is present, called SMN2. SMN2 is retained in all SMA patients and encodes an identical protein compared to SMN1. However, a single silent nucleotide difference in SMN2 exon 7...

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PMID: 16580882
DOI: 10.1016/j.ymthe.2006.01.012

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Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral VectorPMID 16580882Read ↗

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