Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides

Splice-switching antisense oligonucleotides (ASOs), which bind specific RNA-target sequences and modulate pre-mRNA splicing by sterically blocking the binding of splicing factors to the pre-mRNA, are a promising therapeutic modality to treat a range of genetic diseases. ASOs are typically 15–25 nt l...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Scharner, Juergen, Ma, Wai Kit, Zhang, Qian, Lin, Kuan-Ting, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
Molecular Biology
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6954394/
https://ncbi.nlm.nih.gov/pubmed/31802121
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz1132
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