Abstract: Familial dysautonomia (FD, OMIM 223900; also known as HSAN III or Riley-Day syndrome) is the most prevalent form of hereditary sensory and autonomic neuropathy (HSAN; Axelrod et al., 1974). Patients suffering from autonomic and sensory nervous system impairment have no available effective treatment and the average age of death is approximately 24 years. FD is a congenital and progressive disease almost exclusively caused by inheritance of a single nucleotide mutation in the splice acceptor site in intron 20 of the ELP1 (IKBKAP) gene. The germline mutation leads to particularly poor transcript splicing in sympathetic and some sensory neurons which in turn leads to a translational frame shift, introduction of a non-sense codon, and premature truncation and degradation of the encoded Elp1 protein (Anderson et al., 2001; Slaugenhaupt et al., 2001). The resulting low level of Elp1 protein in sympathetic and some sensory neurons leads to their death and results in physiologic sympathetic and sensory nervous system dysfunction (Tourtellotte, 2016). Several laboratories have developed transgenic mice to recapitulate major features of the disease to provide experimental tools to better study disease pathophysiology and potential therapy (Hims et al., 2007; Chen et al., 2009; Jackson et al., 2014; Morini et al., 2016), but how Elp1 functions in disease relevant neurons has not been precisely defined until recently.