How does gene therapy work?
A new gene is inserted into a cell that either corrects a function or imposes on the cell a new function. Genes are unwieldy, since they’re large, highly charged, and need to get to the right cells. If done properly, the gene will take up residence in the cell and persist for the life of the cell, setting the stage for a cure. Viruses are co-opted as gene-delivery vehicles—also called vectors—by exchanging their disease-causing genes with therapeutic genes.
