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Generation of a Gene-corrected Human Induced Pluripotent Stem Cell Line Derived from a Patient with Laterality Defects and Congenital Heart Anomalies with a C.455G > A Alteration in DAND5

Overview
Journal Stem Cell Res
Publisher Elsevier
Specialty Cell Biology
Date 2019 Dec 24
PMID 31869685
Citations 2
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Abstract

Human induced pluripotent stem cells (hiPSCs) from individual patient basis are considered a powerful resource to model human diseases. However, to study complex multigenic diseases such as Congenital Heart Disease, it is crucial to generate perfect isogenic controls to understand gene singularity and contribution. Here, we report the engendering of an isogenic hiPSC line with homozygous correction of c.455G > A alteration in the DAND5 gene, using CRISPR/Cas9 technology. The characterization of a clone of this cell line demonstrates normal karyotype, pluripotent state, and potential to differentiate in vitro towards endoderm, mesoderm, and ectoderm.

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