An iPSC Model of Hereditary Sensory Neuropathy-1 Reveals L-Serine-Responsive Deficits in Neuronal Ganglioside Composition and Axoglial Interactions

Scientists used induced pluripotent stem cells from patients with hereditary sensory neuropathy type 1 to determine whether endogenous 1-deoxysphingolipid bases were neurotoxic, patho-mechanisms of toxicity and response to therapy.