ELP1 and Fabry disease: Our recent work with Ikbkap CKO mice, which lack Ikbkap in both CNS and peripheral nervous system (PNS) neurons, demonstrated that loss of Ikbkap in RGCs causes their progressive death (Ueki et al., 2016), recapitulating the retinal phenotype of the FD patients (Mendoza-Santiesteban et al., 2014, 2017).