Science and Research

Targeted Gene Editing in Human Pluripotent Stem Cells Using Site-Specific Nucleases

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.

  • Merkert, S.; Martin, U.

Keywords

  • CRISPR/Cas9
  • Homologous recombination
  • Nhej
  • Talen
  • Zfn
Publication details
DOI: 10.1007/10_2017_25
Journal: Advances in biochemical engineering/biotechnology
Work Type: Review
Location: BREATH
Disease Area: ROR
Partner / Member: MHH
Access-Number: 29124278

DZL Engagements

chevron-down