Generation of isogenic and homozygous MEN1 mutant cell lines from patient-derived iPSCs using CRISPR/Cas9

使用 CRISPR/Cas9 从患者来源的 iPSC 生成同源和纯合 MEN1 突变细胞系

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作者:Naomi Even-Zohar, Derya Metin-Armagan, Anat Ben-Shlomo, Dhruv Sareen, Shlomo Melmed

Abstract

MEN1, an autosomal dominant disorder caused by mutations in the tumor suppressor gene MEN1, manifests with co-occurrence of multiple endocrine/neuroendocrine neoplasms. An iPSC line derived from an index patient carrying the mutation c.1273C>T (p.Arg465*) was edited using a single multiplex CRISPR/Cas approach to create an isogenic control non-mutated line and a homozygous double mutant line. These cell lines will be useful for elucidating subcellular MEN1 pathophysiology and for screening to identify potential MEN1 therapeutic targets.

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