Back

Engineered Bxb1 variants improve integrase activity and fidelity

Rose, J.; Kim, H.; Von Stetina, J.; Thangavelu, M.; Conway, H.; O'Hara, M. S.; Hanna, P.; Biondi, T.; Dunyak, M. T.; Finn, J. D.; Kumar, S.; Cochrane, J. C.

2024-10-21 biochemistry
10.1101/2024.10.21.619419 bioRxiv
Show abstract

Many current genome editing technologies rely on the action of large serine integrases (LSIs) to insert gene-sized DNA sequences into the genome. Bxb1 is the most commonly used LSI for therapeutic efforts, including PASTE, PASSIGE and I-PGI. While Bxb1 demonstrated good activity in vitro in cycling cells, the activity in non-dividing hepatocytes was significantly less efficient. Further, wild-type Bxb1 is known to have detectable off-target activity at cryptic genomic sites, which presents a potential safety risk for therapeutic development. To address these issues, we developed Bxb1 variants that demonstrate increased specificity and potency in vitro and have engineered stabilized Bxb1 variants that increase in vivo activity over 25-fold enabling targeted integration at therapeutically relevant levels.

Matching journals

The top 2 journals account for 50% of the predicted probability mass.

50% of probability mass above

"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.