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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2018
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5854547/ https://www.ncbi.nlm.nih.gov/pubmed/29858094 http://dx.doi.org/10.1016/j.omtn.2018.01.011 |