CRISPR-Cas systems represent the cutting edge technology for genome editing with a wide range of applications, from basic research science in model organisms to the highest advance therapeutics applications to develop new antimicrobials and to cure genetic diseases. The power of CRISPR technology is based on a reprogrammable nuclease that can be guide to a desire target to cut DNA (or RNA) and repair the mutation as need be. However, the limiting factor is to be able to get the CRISPR systems, usually based on the nuclease Cas9, inside of the cell. Different approaches has been use successfully in the past using attenuate virus that carry Cas9 and inject it in the target cell.
Recently, Lee and coworkers present in Nature*, the last approach to overcome the limiting factor of delivering Cas9, using gold nanoparticles to deliver Cas9 and also the repair template the cell should use to correct the mutation. They proved the functionality of this system to efficiently correct the DNA mutation that causes Duchenne muscular dystrophy in mice.
Again, CRISPR technologies represent the highest revolution in genome editing ever and its potential therapeutic applications in any kind of cell and organism. Some limiting factors are still unsolved, like the best delivery method depending on the cell target location and also the off target affects, but hopefully during the next five years CRISPR application in humans will be real.
*Reference to the original article:
https://www.nature.com/articles/s41551-017-0137-2