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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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Detalles Bibliográficos
Publicado en:Int J Mol Sci
Main Authors: Noé, Véronique, Ciudad, Carlos J.
Formato: Artigo
Idioma:Inglês
Publicado: MDPI 2021
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Acceso en liña: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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