Improved gene editing method could power future cell and gene therapies

A new technique based on special cell-penetrating peptides promises advantages over current methods for editing the genomes of primary cells, such as patients’ T cells.

A new approach to the genetic engineering of cells promises significant improvements in speed, efficiency, and reduction in cellular toxicity compared to current methods. The approach could also power the development of advanced cell therapies for cancers and other diseases, according to a study from researchers in the Perelman School of Medicine.

Microscopic view of DNA strands.
Image: Courtesy of Penn Medicine News

In the study, published in Nature Biotechnology, researchers find that protein fragments used by some viruses to help them get into cells could also be used to get CRISPR-Cas gene editing molecules into cells and their DNA-containing nuclei with extraordinarily high efficiency and low cellular toxicity.

The scientists expect the new technique to be particularly useful for modifying T cells and other cells from a patient’s own body to make cell therapies. One such application could be CAR T (chimeric antigen receptor T cell) therapy, which uses specially modified immune cells from a patient to treat cancer. The T cells—a type of white blood cell—are removed from the patient and reprogrammed to find and attack cancer cells when reintroduced to the bloodstream.

“This new approach—building on Penn Medicine’s history of cell and gene therapy innovation—has the potential to be a major enabling technology for engineered cellular therapies,” says co-senior author E. John Wherry, Richard and Barbara Schiffrin President’s Distinguished Professor and chair of systems pharmacology & translational therapeutics at Penn Medicine.

This story is by Eric Horvath. Read more at Penn Medicine News.