Therapeutic genome editing with engineered nucleases
Haas, S.; Dettmer, V.; Cathomen, T. · Zeitschrift für Orthopädie und Unfallchirurgie · 2017 · Heft 01 · S. 45 bis 52
Bibliografische Angaben
Zusammenfassung
SummaryTargeted genome editing with designer nucleases, such as zinc finger nucleases, TALE nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic disorders, which have not been amenable to conventional gene-addition-type gene therapy approaches, such as disorders with dominant inheritance or diseases caused by mutations in tightly regulated genes, can now be treated by precise genome surgery. Moreover, engineered nucleases enable novel genetic interventions to fight infectious diseases or to improve cancer immunotherapies. Here, we review the development of the different classes of…