Article

Towards a combined therapy for spinal muscular atrophy based on opposing effects of an antisense oligonucleotide on chromatin and splicing

2021-09-24

Abstract excerpt

<h4>Summary</h4> Spinal Muscular Atrophy (SMA) is a motor-neuron disease caused by loss-of-function mutations of the SMN1 gene. Humans have a paralog, SMN2 , whose exon 7 is predominantly skipped, and so it cannot fully compensate for the lack of SMN1 . Nusinersen (Spinraza) is a splicing-correcting antisense oligonucleotide drug (ASO) approved for clinical use. Nusinersen targets a splicing silencer located i...

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Literature Corpus work: 3677e186-53de-5864-87fe-60c5ff6541d6
DOI: 10.1101/2021.09.24.461646

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