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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Detalles Bibliográficos
Autores principales: Wijaya, Yogik Onky Silvana, Niba, Emma Tabe Eko, Nishio, Hisahide, Okamoto, Kentaro, Awano, Hiroyuki, Saito, Toshio, Takeshima, Yasuhiro, Shinohara, Masakazu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9027857/
https://www.ncbi.nlm.nih.gov/pubmed/35456491
http://dx.doi.org/10.3390/genes13040685

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9027857/
https://www.ncbi.nlm.nih.gov/pubmed/35456491
http://dx.doi.org/10.3390/genes13040685

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