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Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

While most monogenic diseases are caused by loss or reduction of protein function, the need for technologies that can selectively increase levels of protein in native tissues remains. Here we demonstrate that antisense-mediated modulation of pre-mRNA splicing can increase endogenous expression of fu...

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Detalles Bibliográficos
Autores principales: Lim, Kian Huat, Han, Zhou, Jeon, Hyun Yong, Kach, Jacob, Jing, Enxuan, Weyn-Vanhentenryck, Sebastien, Downs, Mikaela, Corrionero, Anna, Oh, Raymond, Scharner, Juergen, Venkatesh, Aditya, Ji, Sophina, Liau, Gene, Ticho, Barry, Nash, Huw, Aznarez, Isabel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7347940/
https://www.ncbi.nlm.nih.gov/pubmed/32647108
http://dx.doi.org/10.1038/s41467-020-17093-9