Antisense Technology

Investigators at University of Missouri target antisense technology

Published in Gene Therapy Weekly, February 12th, 2009

"Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder and is the leading genetic cause of infant mortality. SMA is caused by the loss of survival motor neuron-1 (SMN1)," scientists writing in the journal Human Gene Therapy report.

"In humans, a nearly identical copy gene is present called SMN2, but this gene cannot compensate for the loss of SMN1 because of a single silent nucleotide difference in SMN2 exon 7. This single-nucleotide difference attenuates an exonic splice enhancer, resulting in the production of an alternatively spliced isoform lacking exon 7, which is essential for protein function. SMN2, however, is a critical disease...

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Source: Gene Therapy Weekly (2009-02-12)

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