The pathogenic pseudoexons created by deep-intronic SAVs can be pharmacologically targeted, as we previously demonstrated in two pathological models: the IVS4 + 866 C > T causative variant of NEMO of anhidrotic ectodermal dysplasia with immunodeficiency, and the c.3849 + 10kbC > T of CFTR of cystic fibrosis [43, 44]. Here, CFTR is linked to immune system disorder.