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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Detalles Bibliográficos
Autores principales: Flynn, Loren L., Mitrpant, Chalermchai, Pitout, Ianthe L., Fletcher, Sue, Wilton, Steve D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
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

Internet

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

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