To the Editor: Genome editing via programmable endonucleases enables us to generate site-specific double-strand breaks at virtually any position in a target genome.1,2,3 Exploiting cellular repair mechanisms, this can be used for targeted gene disruption via nonhomologous end joining (NHEJ) or for the precise manipulation of a target sequence through homology-directed repair (HDR) in the presence of a suitable DNA template. The latter carries great promise for the field of gene therapy as it can be utilized for the correction of disease-causing mutations.
No takes yet. Share an insight, caveat, or question.
Glaser et al. (2016) studied this question.
Synapse has enriched 2 closely related papers on similar clinical questions. Consider them for comparative context: