Genome editing technologies also have the potential in the future to be utilised as a therapeutic agent in their own right, and to repair the genetic mutations contributing to disease (Cox et al. 2015). Such reagents could not only be applied in an in vivo context, but also to repair causative lesions in patient-derived iPSCs that could subsequently be used to generate specific cell types to use as cellular therapies. Such strategies have shown significant promise in some cases, for example in the treatment of a chemically induced primate model of Parkinson’s disease through injection of autologous iPSC-derived dopaminergic neurons (Emborg et al. 2013; Hallett et al. 2015). Therapies for HIV (Tebas et al. 2014) and cancer (Fesnak et al. 2016) involving genome editing are already in clinical trials, and the next few years will likely herald exciting developments in this area of somatic gene therapy.
