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

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

In collaboration with Dr Chalermchai Mitrpant, Charoensawan laboratory at Department of Biochemistry, Faculty of Science, has contributed to the transcriptomic analyses showing that splice switching antisense oligonucleotides could redirect the splicing repressor hnRNPA1 to the hnRNPA1b isoform and restore SMN expression in fibroblasts from a patient with type I SMA, a severe, debilitating neuromuscular condition characterised by loss of motor neurons and progressive muscle wasting.

References
Toosaranont J, Ruschadaariyachat S, Mujchariyakul W, Arora JK, Charoensawan V, Suktitipat B, Palmer TN, Fletcher S, Wilton SD, Mitrpant C. Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts. Int J Mol Sci. 2022 Apr 1;23(7):3937.

DOI: https://doi.org/10.3390/ijms23073937