Original paper
SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice
Abstract
Spinal muscular atrophy (SMA), which results from the loss of expression of the survival of motor neuron-1 (SMN1) gene, represents the most common genetic cause of pediatric mortality. A duplicate copy (SMN2) is inefficiently spliced, producing a truncated and unstable protein. We describe herein a potent, orally active, small-molecule enhancer of SMN2 splicing that elevates full-length SMN protein and extends survival in a severe SMA mouse...
Paper Details
Title
SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice
Published Date
Jun 1, 2015
Journal
Volume
11
Issue
7
Pages
511 - 517
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Notes
History