Background

Targeted genome modification provides a powerful tool for the development of new therapies for human genetic diseases. Several approaches for the targeted editing of genomes are available such as Zinc-Finger Nucleases (ZFNs), Transcription factor Activator-like Nucleases (TALENs) or Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) nucleases. However, these methods have limitations, such as being not applicable for high-throughput editing (ZFNs, TALENs) or having constraints for a specific sequence in order to be functional (CRISPR nucleases require a Proto-spacer adjacent motif, or PAM).