HyEdit - A Tool for Precision Genome Editing
An artificial system for precise genome editing which is suitable for therapeutic and diagnostic applications
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).
Technology Overview
With HyEdit (Hybridization-based precision genome cleavage and EDITing), we present an artificial system for precise genome editing which is suitable for therapeutic and diagnostic applications. The approach is based on an RNA-guided nuclease, independent of CRISPR nucleases. The HyEdit system can target both, DNA and RNA, and is highly customizable. Replacing the nuclease domain of HyEdit with a transcriptional or translational effector enables control over gene expression, akin to CRISPR interference and CRISPR activation. Replacing the nuclease domain of HyEdit with fluorophores further enables in vivo labelling of specific DNA and RNA loci, as well as in vitro diagnostics (e.g. viral RNA detection).
Benefits
HyEdit overcomes current limitations in genome editing by being fully independent of sequence motifs and having high specificity even for highly repetitive genomic regions by extending the length of hybridizing RNAs (hyRNAs). In addition, the protein component of HyEdit is very small, even compared to minimized CRISPR nucleases, which allows for adenovirus packaging, as well as facilitated localization into organelle compartments to enable e.g. editing of mitochondrial or chloroplast DNA.
Opportunity
The technology is available for out-licensing or co-development. Additionally a technology evaluation program is also on offer.