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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...
Autores principales: | Ma, Shuangyu, Viola, Ryan, Sui, Lina, Cherubini, Valentino, Barbetti, Fabrizio, Egli, Dieter |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6294262/ https://www.ncbi.nlm.nih.gov/pubmed/30503261 http://dx.doi.org/10.1016/j.stemcr.2018.11.006 |
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