Researchers use our
technologies in their quest to
cure diseases like cancer,
HIV, and Parkinson’s
Insertion of Sequences into Safe-Harbor Loci
Safe-harbor loci are regions of the genome that can be disrupted in a wide variety of cell types without causing adverse effects. These safe-harbor loci are thus ideal sites for inserting exogenous nucleic-acid sequences.
Our scientists developed a method for inserting sequences in safe harbor loci using mRNA encoding gene-editing proteins.
Insertion of Sequences into Safe-Harbor Loci is protected by two pending U.S. patents (with additional patents pending in other countries).
- Achieve high-efficiency insertion into the AAVS1 safe-harbor locus
- Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
- Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to various cells and tissues ex vivo and in vivo