Goodwin, M.; Lee, E.; Lakshmanan, U.; Shipp, S.; Froessl, L.; Barzaghi, F.; Passerini, L.; Narula, M.; Sheikali, A.; Lee, Ciaran M.; Bao, G.; Bauer, C. S.; Miller, H. K.; Garcia-Lloret, M.; Butte, M. J.; Bertaina, A.; Shah, A.; Pavel-Dinu, M.; Hendel, A.; Porteus, M.; Roncarolo, M. G.; Bacchetta, R.
(American Association for the Advancement of Science, 2020-05-06)
The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with limited treatment options. IPEX syndrome is caused by mutations ...