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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...
Autores principales: | , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
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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 |