early onset gene (BRCA1), in which a natural mutation in an ESE in exon 18 causes the exclusion of this exon from the mRNA. The addition of an ESSENCE compound with the exon-18-specific antisense ...

A mutation is any detectable and heritable change ... are considered promising candidates for treatment with antisense oligonucleotides. Here, the authors present a novel framework to screen ...

This project is focused on identifying exons within the NF1 gene that can be skipped while still maintaining function of the gene, allowing these mutations to be bypassed. We have made good progress ...