Targeted Genome Engineering

Christien Bednarski, Sylwia Bobis-Wozowicz, Toni Cathomen, Anne-Kathrin Dreyer, Nico Jäschke, Fabienne Lütge, Kafaitullah Khan, Claudio Mussolino, Shamim Rahman

The goal of the "Targeted genome engineering" lab is to characterize the mechanisms regulating the repair of DNA double-strand breaks (DSBs) and to exploit this knowledge to specifically alter the genetic makeup of cells. Our rational genome editing approach is based on the use of designer nucleases, such as zinc-finger nucleases (ZFNs) or TALE nucleases (TALENs), which we employ to insert a site-specific DSB in the target locus. Depending on which cellular DSB repair pathway is harnessed, ZFNs and TALENs can be used to disrupt genes via the non-homologous end-joining (NHEJ) pathway or to correct mutations in the genome by homologous recombination (HR). We introduce specific alterations in the genome of somatic stem cells with the aim to develop novel treatment options for patients suffering from hereditary disorders and/or to improve our understanding of DNA repair in somatic stem cells.

Selected own references: Händel et al. (2009) Mol Ther 17, 104-11; Maeder et al. (2008) Mol Cell 31, 294-301; Cornu et al. (2008) Mol Ther 16, 352-8; Cornu et al. (2007) Mol Ther 15, 2107-13; Szczepek et al. (2007) Nat Biotechnol 25, 786-93.

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