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Updated: Feb 19, 2026

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
Targeted Gene Editing in Human Pluripotent Stem Cells Using Site-Specific Nucleases
Sylvia Merkert1,2,3, Ulrich Martin4,5,6
1Department of Cardiothoracic, Transplantation and Vascular Surgery, Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Hannover, Germany.
Abstract:
Introduction of induced pluripotent stem cell (iPSC) technology and site-directed nucleases brought a major breakthrough in the development of regenerative therapies and biomedical research. With the advancement of ZFNs, TALENs, and the CRISPR/Cas9 technology, straightforward and precise manipulation of the genome of human pluripotent stem cells (PSC) became possible, allowing relatively easy and fast generation of gene knockouts, integration of transgenes, or even introduction of single nucleotide changes for correction or introduction of disease-specific mutations. We review current applications of site-specific nucleases in human PSCs and focus on trends and challenges for efficient gene editing and improvement of targeting strategies. Graphical Abstract.
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