Scientists from Genzyme Corporation and ISIS Pharmaceuticals Publish Paper Showing Antisense Oligonucleoties (ASOs) Ameliorate Symptoms in a Severe Mouse Model of Spinal Muscular Atrophy
Science Translational Medicine (via FSMA) | March 4, 2011
It was reported in the journal Science Translational Medicine that ASOs designed to correct splicing of the SMN2 gene improve symptoms in a severe mouse model of Spinal Muscular Atrophy when delivered directly to the CNS.
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to treat SMA is to use antisense oligonucleotides (ASOs) to redirect the splicing of the SMN2 gene to boost production of functional SMN.
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