Precise cut-and-paste DNA insertion using engineered type V-K CRISPR-associated transposases
Genome editing technologies capable of generating large sequence insertions would obviate the need to develop custom patient-specific approaches, enabling the treatment of larger swaths of patients with diverse mutations using a single therapeutic. Towards this goal, CGM Investigators led by Ben Kleinstiver recently developed several engineered versions of CRISPR-associated transposases (CASTs) with improved properties that can insert large kilobase-scale DNA cargos into genomes. We engineered CAST enzymes that have dramatically improved safety by reducing their off-target genome-wide integrations, that have enhanced insertion purity and efficiency, and that function for the first time in human cells, positioning CASTs as a leading technology for kilobase-scale genome edits for a new class of genetic medicines.
Read more in Nature.
The Center for Genomic Medicine (CGM) comprises one of the largest and most vibrant hubs of genomic medicine research in the world. The CGM includes 46 faculty collaborating to define the ‘genomic medicine cycle’ – which envisages a genomics community seeking to advance research from basic genomics research to ultimately using genome information for diagnostics and targeted therapeutics. All of our investigators are faculty at Harvard Medical School, and many members of our community are also investigators at the Broad Institute of Harvard and MIT.
