Supplementary Materialsijms-19-00762-s001. we explored was not the skipping of a disease-associated exon, but rather the enhancement of correction, together with an antisense RNA molecule that specifically clogged the competing target region. Dominant mutations in are responsible for the generalized severe form of epidermolysis bullosa simplex (EBS-gen sev). Incorporation of the mutant K14 into the intermediate… Continue reading Supplementary Materialsijms-19-00762-s001. we explored was not the skipping of a disease-associated