Projektnummer

0057181

Zusammenfassung

The underlying reason of inherited malignancies, as well as of some infectious diseases, is based on the alteration (e.g. mutation, DNA-insertion) of the human genome. Current experimental gene therapies substitute mutated inactive genes by their respective wildtype (functional) gene homologues. However, subtle genome alterations, including the faithful removal of deleterious sequences such as inserted viral genomes remain to be a major and so far unsolved technical challenge. Novel designer enzymes, such as zinc-finger nucleases (ZFNs) or transcription activator-like effector nucleas...