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

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. Differentiation of mut...

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Bibliografiske detaljer
Udgivet i:Stem Cell Reports
Main Authors: Ma, Shuangyu, Viola, Ryan, Sui, Lina, Cherubini, Valentino, Barbetti, Fabrizio, Egli, Dieter
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6294262/
https://ncbi.nlm.nih.gov/pubmed/30503261
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2018.11.006
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