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 U.S. Patent Number 10,724,053 (with additional patents pending in the U.S. and in other countries). Of note, certain claims of the granted patent are not limited by type of nucleic acid or formulation.

Example Applications

  • 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