Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
Nucleic acid therapeutics allow sequence-based targeting of disease genes, such as the genes involved in spinal muscular atrophy (SMA) pathogenesis. 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, SMN...
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| Format: | Thesis |
| Language: | English |
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2020
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