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Genomics & Bioinformatics

Designer nucleases allow to target and cleave a specific region within the DNA to either knock out or repair a gene. Even though those designer nucleases are highly specific, off-target activity has been reported, where DNA regions were cleaved with sequences similar to the intended on-target. If off-target cleavage occurs within a gene or regulatory region, unexpected side effects of the event can be envisioned, which in the worst case scenario are tumorigenic. Designing nucleases for novel gene therapeutic approaches and translating them to the clinic, complications by such off-target events must be avoided. Therefore the quality of each designer nuclease has to be validated and possible off-targets be identified. To this end cells are treated with a given nuclease and off-targets are assessed by high throughput sequencing (next generation sequencing, NGS) and the results (big data) are evaluated bioinformatically.


Dr. Julia Klermund

Institute for Transfusion Medicine and Gene Therapy
at Center for Translational Cell Research
Breisacher Straße 115
79106 Freiburg
Phone: 0761-27077747