
Over the last two decades, exciting tools have emerged that allow researchers to cut and paste specific sequences of DNA within living cells, a process called gene editing. These tools, including one adapted from a bacterial defense system called CRISPR, have energized the research community with the possibility of using them to modify human genes associated with disease.
A major barrier to testing medical applications of gene editing has been getting the proteins that do the cutting into the cells of living animals. The main methods used in the laboratory take a roundabout route: Researchers push the DNA templates for making the proteins into cells, and then the cells’ own protein factories produce the editing proteins.
Researchers led by David Liu from Harvard University are trying to cut out the middleman, so to speak, by ferrying the editing proteins, not the DNA instructions, directly into cells. In a proof-of-concept study, their system successfully delivered three different types of editing proteins into cells in the inner ears of live mice. Continue reading “Delivering Gene-Editing Proteins to Living Cells”