Although ASO cannot be applied in case of a homozygous NPHP1 deletion which is the most common cause of NPH, it can be used to target downstream dysregulated genes as was demonstrated for Agt encoding angiotensinogen in the treatment of Pkd1 mice (Saigusa et al., 2016; Fitzgibbon et al., 2018). This evidence concerns the gene NPHP1 and normal pressure hydrocephalus.