Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers

Exon skipping uses chemically modified antisense oligonucleotides to modulate RNA splicing. Therapeutically, exon skipping can bypass mutations and restore reading frame disruption by generating internally truncated, functional proteins to rescue the loss of native gene expression. Limb-girdle muscu...

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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Wyatt, Eugene J., Demonbreun, Alexis R., Kim, Ellis Y., Puckelwartz, Megan J., Vo, Andy H., Dellefave-Castillo, Lisa M., Gao, Quan Q., Vainzof, Mariz, Pavanello, Rita C. M., Zatz, Mayana, McNally, Elizabeth M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2018
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6012523/
https://ncbi.nlm.nih.gov/pubmed/29720576
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.99357
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