Targeted knock-ins with pseudovirus for the stable expression of large transgenes.

Nat Biomed Eng · 2023

basic_science · Level V

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Abstract

We engineered integrase-deficient lentiviruses to act as vectors for the delivery of large gene knock-ins via homology-directed repair. This technology enables the non-cytotoxic, targeted insertion of difficult-to-express transgenes into genomic loci that are essential to cell survival, thereby overcoming the gene silencing that otherwise limits primary immune cell engineering.