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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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Sci Rep
Päätekijät: Chen, Suxiang, Le, Bao T., Chakravarthy, Madhuri, Kosbar, Tamer R., Veedu, Rakesh N.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group UK 2019
Aiheet:
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC6465270/
https://ncbi.nlm.nih.gov/pubmed/30988454
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-42523-0
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