Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of <i>SMN2</i> in SMA Type I Fibroblasts

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 <i>SMN1</i> that encodes the survival motor neuron (SMN) protein necessary for the survival of m...

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Main Authors: Jarichad Toosaranont, Sukanya Ruschadaariyachat, Warasinee Mujchariyakul, Jantarika Kumar Arora, Varodom Charoensawan, Bhoom Suktitipat, Thomas N. Palmer, Sue Fletcher, Steve D. Wilton, Chalermchai Mitrpant
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:International Journal of Molecular Sciences
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Online Access:https://www.mdpi.com/1422-0067/23/7/3937