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Article

Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides

11 Oct 2019

Abstract excerpt

The process of pre-mRNA splicing is a common and fundamental step in the expression of most human genes. Alternative splicing, whereby different splice motifs and sites are recognised in a developmental and/or tissue-specific manner, contributes to genetic plasticity and diversity of gene expression. Redirecting pre-mRNA processing of various genes has now been validated as a viable clinical therapeutic strategy,...

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PMID: 31614438
DOI: 10.3390/ijms20205030

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