Abstract
Programmable nucleases, such as zinc finger nucleases, TALE nucleases, and CRISPR-Cas nucleases, allow for the introduction of precise modifications in the human genome. As a consequence, targeted genome editing has heralded a new area for gene therapy and the treatment of acquired and inherited disorders. Here, we describe the concept of designer nuclease- mediated genome engineering, discuss risks and potential limitations of its usage, highlight some of the achieved milestones, and review the most prominent studies that have made it from the laboratory to the clinic.
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Viviane Dettmer 2009–2014 Bachelor- und Masterstudium in Human- und Molekularbiologie an der Universität des Saarlandes. 2014–2015 ASTAR Trainee Scholarship Singapore. Seit 2015 Doktorandin am Institut für Zell- und Gentherapie und am Centrum für Chronische Immundefizienz, Universitätsklinikum Freiburg.
Toni Cathomen 1998 Promotion an der Universität Zürich, Schweiz. 1999–2003 Postdoktorat am Salk Institute in San Diego, CA, USA. 2003–2009 W1-Professur an der Charité in Berlin. 2009–2012 W2-Professur an der Medizinischen Hochschule Hannover. Seit 2012 W3-Professor und Direktor des Instituts für Zell- und Gentherapie am Universitätsklinikum Freiburg.
Markus Hildenbeutel 2006 Diplom in Biologie an der Universität Heidelberg. 2007–2011 Promotion an der TU Kaiserslautern. 2011–2014 Postdoktorandenstipendium der Wenner-Gren-Stiftung, Universität Stockholm, Schweden. Seit 2015 Postdoktorand am Institut für Zell- und Gentherapie und am Centrum für Chronische Immundefizienz, Universitätsklinikum Freiburg.
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Dettmer, V., Cathomen, T. & Hildenbeutel, M. Genom-Editierung — neue Wege im klinischen Alltag. Biospektrum 23, 155–158 (2017). https://doi.org/10.1007/s12268-017-0781-9
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DOI: https://doi.org/10.1007/s12268-017-0781-9