Science and Research

Site-Specific Genome Engineering in Human Pluripotent Stem Cells

The possibility to generate patient-specific induced pluripotent stem cells (iPSCs) offers an unprecedented potential of applications in clinical therapy and medical research. Human iPSCs and their differentiated derivatives are tools for diseases modelling, drug discovery, safety pharmacology, and toxicology. Moreover, they allow for the engineering of bioartificial tissue and are promising candidates for cellular therapies. For many of these applications, the ability to genetically modify pluripotent stem cells (PSCs) is indispensable, but efficient site-specific and safe technologies for genetic engineering of PSCs were developed only recently. By now, customized engineered nucleases provide excellent tools for targeted genome editing, opening new perspectives for biomedical research and cellular therapies.

  • Merkert, S.; Martin, U.

Keywords

  • *CRISPR-Cas Systems
  • Gene Editing/*methods
  • Genetic Therapy/*methods
  • Humans
  • Induced Pluripotent Stem Cells/*metabolism
  • clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9
  • human iPSCs
  • targeted genome engineering
  • transcription activator-like effector nuclease (TALEN)
  • zinc-finger nucleases (ZFNs)
Publication details
DOI: 10.3390/ijms17071000
Journal: International journal of molecular sciences
Number: 7
Work Type: Review
Location: BREATH
Disease Area: ROR
Partner / Member: MHH
Access-Number: 27347935
See publication on PubMed

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