High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts
Spinal muscular atrophy (SMA) is caused by survival motor neuron 1 SMN1 deletion. The survival motor neuron 2 (SMN2) encodes the same protein as SMN1 does, but it has a splicing defect of exon 7. Some antisense oligonucleotides (ASOs) have been proven to correct this defect. One of these, nusinersen...
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| Main Authors: | , , , , , , , |
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| Format: | Article PeerReviewed |
| Language: | English |
| Published: |
MDPI
2022
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| Subjects: | |
| Online Access: | https://repository.ugm.ac.id/279049/1/High-Concentration-or-Combined-Treatment-of-Antisense-Oligonucleotides-for-Spinal-Muscular-Atrophy-Perturbed-SMN2-Splicing-in-Patient-FibroblastsGenes.pdf https://repository.ugm.ac.id/279049/ https://www.mdpi.com/2073-4425/13/4/685 https://doi.org/10.3390/genes13040685 |
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| Institution: | Universitas Gadjah Mada |
| Language: | English |