Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts
Spinal muscular atrophy (SMA) is a severe, debilitating neuromuscular condition char-acterised by loss of motor neurons and progressive muscle wasting. SMA is caused by a loss of expression of SMN1 that encodes the survival motor neuron (SMN) protein necessary for the survival of motor neurons. Rest...
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Main Authors: | , , , , , , , , , |
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Format: | Article |
Published: |
2022
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Online Access: | https://repository.li.mahidol.ac.th/handle/123456789/73372 |
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Institution: | Mahidol University |