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Insertion of Sequences into Safe-Harbor Loci

Description

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).


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