Like any new technology that has been decades in the making, gene therapy is evolving. The classical approach for in vivo gene therapies involves the delivery of additional genetic information to particular cells in order to express a protein that will be curative in a given disease state. With the persistence of notable challenges such as vector safety issues, tissue targeting and durability, there is intense selection pressure on the technologies employed. Newer approaches to gene therapy are garnering a lot of attention, being a standout theme at the annual J.P. Morgan Healthcare Conference in January 2022. Ahead of a catalyst-rich year to come, this article serves as a primer for the various editing technologies and players. Should their potential be fulfilled, then gene editing will hasten the demise of earlier and inferior generations of cell and gene therapy platforms.
Since the first publications in the early 2010s, gene editing has gained rapid prominence and recognition for its utility across the life sciences. CRISPR pioneers Professors Charpentier and Doudna shared...
