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Polypurine Reverse-Hoogsteen Hairpins as a Tool for Exon Skipping at the Genomic Level in Mammalian Cells

Therapeutic strategies for rare diseases based on exon skipping are aimed at mediating the elimination of mutated exons and restoring the reading frame of the affected protein. We explored the capability of polypurine reverse-Hoogsteen hairpins (PPRHs) to cause exon skipping in NB6 cells carrying a...

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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Noé, Véronique, Ciudad, Carlos J.
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2021
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8038689/
https://ncbi.nlm.nih.gov/pubmed/33917446
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22073784
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