title: Site-Specific Genome Engineering in Human Pluripotent Stem Cells.
abstract: 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.
authors.affiliation: Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, Hannover Medical School, 30625 Hannover, Germany. merkert.sylvia@mh-hannover.de.|Regenerative Biology and Reconstructive Therapies (REBIRTH), Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany. merkert.sylvia@mh-hannover.de.|Biomedical Research in Endstage and Obstructive Lung Disease (BREATH), Member of the German Center for Lung Research (DZL), Hannover Medical School, 30625 Hannover, Germany. merkert.sylvia@mh-hannover.de.
authors.forename: Sylvia
authors.initials: S
authors.lastname: Merkert
authors.affiliation: Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, Hannover Medical School, 30625 Hannover, Germany. martin.ulrich@mh-hannover.de.|Regenerative Biology and Reconstructive Therapies (REBIRTH), Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany. martin.ulrich@mh-hannover.de.|Biomedical Research in Endstage and Obstructive Lung Disease (BREATH), Member of the German Center for Lung Research (DZL), Hannover Medical School, 30625 Hannover, Germany. martin.ulrich@mh-hannover.de.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Martin
mesh_terms: D064113:CRISPR-Cas Systems; D000072669:Gene Editing / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000378:metabolism*
keywords: clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9; human iPSCs; targeted genome engineering; transcription activator-like effector nuclease (TALEN); zinc-finger nucleases (ZFNs)