Browsing by Author "Wilton, SD"
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Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts.
Toosaranont, J; Ruschadaariyachat, S; Mujchariyakul, W; Arora, JK; Charoensawan, V; Suktitipat, B; Palmer, TN; Fletcher, S; Wilton, SD; Mitrpant, C (2022-04-01)Spinal muscular atrophy (SMA) is a severe, debilitating neuromuscular condition characterised by loss of motor neurons and progressive muscle wasting. SMA is caused by a loss of expression of SMN1 that encodes the survival ...