DNA Editing

A New Class of Medicines through DNAEditing
Matthew H. Porteus, M.D., Ph.D.

Genome editing is a set of methods used to change the DNA of a cell with single base-pair precision. It is a specific form of gene therapy, and the engineering of cells through genome editing has the potential to create a new class of medicines for the treatment of both genetic and nongenetic diseases. Genome editing has entered clinical trials: applications include the correction of variants that cause monogenic diseases, the enhancement of chimeric antigen receptor (CAR) T-cell therapy, and cell-based regenerative medicine. Here I describe the development of genome editing and discuss the ways in which efficacy, specificity, delivery, and safety are integral to this process (see interactive graphic, available at NEJM.org).