Generation of an ASS1 heterozygous knockout human embryonic stem cell line, WAe001-A-13, using CRISPR/Cas9


The ASS1 gene encodes argininosuccinate synthetase-1, a cytosolic enzyme with a critical role in the urea cycle. Mutations are found in all ASS1 exons and cause the autosomal recessive disorder citrullinemia. Using CRISPR/Cas9-editing, we established the WAe001-A-13 cell line, which was heterozygous for an ASS1 mutation, from the human embryonic stem cell line H1. The WAe001-A-13 cell line maintained the pluripotent phenotype, the ability to differentiate into all three germ layers and a normal karyotype. Copyright © 2017. Published by Elsevier B.V.

Authors Yuan F, Guo D, Liu Y, Xu Y, Gao G, Wu Y, Yang F, Ke X, Lai K, Wei H, Li YX
Journal Stem cell research
Publication Date 2018 Jan;26:67-71
PubMed 29247816
DOI 10.1016/j.scr.2017.11.007

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