Targeting the 5’ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
(2021)
Journal Article
Bowerman. (2021). Targeting the 5’ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy. Molecular Therapy - Nucleic Acids, 731-742. https://doi.org/10.1016/j.omtn.2020.12.027
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 gene (SMN1). All patients have at least one copy of a paralog, SMN2, but a C-to-T transition in this gene results in exon 7 skipping in a maj... Read More about Targeting the 5’ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy.