Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

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Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (<i>SMN2</i>) gene. All patients have at least one copy of a paralog, <i>SMN2</i>, but a C-to-T transition in this gene results in exon 7 skipping in a majority of tra...

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Hlavní autoři: Winkelsas, AM, Grunseich, C, Harmison, GG, Chwalenia, K, Rinaldi, C, Hammond, SM, Johnson, K, Bowerman, M, Arya, S, Talbot, K, Wood, MJ, Fischbeck, KH
Médium: Journal article
Jazyk:English
Vydáno: Cell Press 2021
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