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.

June 21, 2023


CGM Primary Investigator