Article
Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy.
Proceedings of the National Academy of Sciences of the United States of America - 27 Oct 2015
Bogdanik Laurent P, Osborne Melissa A, Davis Crystal, Martin Whitney P, Austin Andrew, Rigo Frank, Bennett C Frank, Lutz Cathleen M
Abstract excerpt
Clinical presentation of spinal muscular atrophy (SMA) ranges from a neonatal-onset, very severe disease to an adult-onset, milder form. SMA is caused by the mutation of the Survival Motor Neuron 1 (SMN1) gene, and prognosis inversely correlates with the number of copies of the SMN2 gene, a human-specific homolog of SMN1. Despite progress in identifying potential therapies for the treatment of SMA, many questions...
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