β Cell Replacement after Gene Editing of a Neonatal Diabetes-Causing Mutation at the Insulin Locus

Summary: Permanent neonatal diabetes mellitus (PNDM) can be caused by insulin mutations. We generated induced pluripotent stem cells from fibroblasts of a patient with PNDM and undetectable insulin at birth due to a homozygous mutation in the translation start site of the insulin gene. Differentiati...

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Bibliographic Details
Main Authors: Shuangyu Ma, Ryan Viola, Lina Sui, Valentino Cherubini, Fabrizio Barbetti, Dieter Egli
Format: Article
Language:English
Published: Elsevier 2018-12-01
Series:Stem Cell Reports
Online Access:http://www.sciencedirect.com/science/article/pii/S2213671118304740