Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro

Antisense oligonucleotide (AO)-mediated splice modulation has been established as a therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to correct splicing defects in the dystrophin gene was approved by the US Food and Drug Administration (FDA) for the treatment...

Descripción completa

Detalles Bibliográficos
Autores principales: Chen, Suxiang, Le, Bao T., Chakravarthy, Madhuri, Kosbar, Tamer R., Veedu, Rakesh N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6465270/
https://www.ncbi.nlm.nih.gov/pubmed/30988454
http://dx.doi.org/10.1038/s41598-019-42523-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6465270/
https://www.ncbi.nlm.nih.gov/pubmed/30988454
http://dx.doi.org/10.1038/s41598-019-42523-0

Ejemplares similares