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
Summary
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. Copyright © 2019 The Author(s). Published by Elsevier B.V. All rights reserved.
| Authors | Inácio JM, Almeida M, Cristo F, Belo JA |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2020 Jan;42:101677 |
| PubMed | 31869685 |
| DOI | 10.1016/j.scr.2019.101677 |