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Cell Stem Cell
Published

Recapitulation of endogenous 4R tau expression and formation of insoluble tau in directly reprogrammed human neurons.

Authors

Lucia S Capano, Chihiro Sato, Elena Ficulle, Anan Yu, Kanta Horie, Ji-Sun Kwon, Kyle F Burbach, Nicolas R Barthélemy, Susan G Fox, Celeste M Karch, Randall J Bateman, Henry Houlden, Richard I Morimoto, David M Holtzman, Karen E Duff, Andrew S Yoo

Abstract

Tau is a microtubule-binding protein expressed in neurons, and the equal ratios between 4-repeat (4R) and 3-repeat (3R) isoforms are maintained in normal adult brain function. Dysregulation of 3R:4R ratio causes tauopathy, and human neurons that recapitulate tau isoforms in health and disease will provide a platform for elucidating pathogenic processes involving tau pathology. We carried out extensive characterizations of tau isoforms expressed in human neurons derived by microRNA-induced neuronal reprogramming of adult fibroblasts. Transcript and protein analyses showed that miR neurons expressed all six isoforms with the 3R:4R isoform ratio equivalent to that detected in human adult brains. Also, miR neurons derived from familial tauopathy patients with a 3R:4R ratio altering mutation showed increased 4R tau and the formation of insoluble tau with seeding activity. Our results collectively demonstrate the utility of miRNA-induced neuronal reprogramming to recapitulate endogenous tau regulation comparable with the adult brain in health and disease.

PMID:35659876 | DOI:S1934-5909(22)00200-4