Adapting RNA-guided Transposition Systems for Genome Editing in Eukaryotes

Moonshot Award

Patent or Technology Development

Abstract

Current CRISPR-based gene therapies have demonstrated gene therapy as a viable tool for curing previously uncurable genetic diseases, however, current technologies are limited in the size of the edits they can make, low efficiency editing in certain cell types, and unintended off-target mutations. There exists an unmet need for tools that can introduce large edits, function in all cell types, and reduce the risk of unintended mutations. 

The I-F3 CAST family of Tn7 DNA transposons use RNA-guided CRISPR machinery to direct DNA integration to a specific location, can insert large cargo of DNAs, and do not require host cell DNA repair machinery to facilitate their editing.