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Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript
The severe childhood disease spinal muscular atrophy (SMA) arises from the homozygous loss of the survival motor neuron 1 gene (SMN1). A homologous gene potentially encoding an identical protein, SMN2 can partially compensate for the loss of SMN1; however, the exclusion of a critical exon in the cod...
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| Gepubliceerd in: | Mol Ther Nucleic Acids |
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| Hoofdauteurs: | , , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Society of Gene & Cell Therapy
2018
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5854547/ https://ncbi.nlm.nih.gov/pubmed/29858094 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.01.011 |
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