Jocelyn Kaiser, Science
While gene editing techniques such as CRISPR/Cas9 have changed the way basic research is being conducted, widespread use of the technology in medical treatments could be years away. As with previous promising gene therapies the largest obstacle is delivery of the required proteins into the organs of interest. The repair of the mutations behind genetic disorders often necessitates the use of HDR, however as HDR has very low efficiencies most of the treated cells may end up being repaired by NHEJ, presenting a challenging obstacle to researchers. While many gene therapists are excited for the potential of CRISPR/Cas9 technology, years of research are needed before it sees clinical application.