Fig. 1 | Signal Transduction and Targeted Therapy

Fig. 1

From: Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

Fig. 1

Genome editing platforms and mechanisms for DSB repair with endogenous DNA. Genome editing nucleases (ZFNs, TALENs and CRISPR/Cas9) induce DSBs at targeted sites. DSBs can be repaired by NHEJ or, in the presence of donor template, by HDR. Gene disruption by targeting the locus with NHEJ leads to the formation of indels. When two DSBs target both sides of a pathogenic amplification or insertion, a therapeutic deletion of the intervening sequences can be created, leading to NHEJ gene correction. In the presence of a donor-corrected HDR template, HDR gene correction or gene addition induces a DSB at the desired locus. DSB double-stranded break, ZFN zinc-finger nuclease, TALEN transcription activator-like effector nuclease, CRISPR/Cas9 clustered regularly interspaced short palindromic repeat associated 9 nuclease, NHEJ nonhomologous end-joining, HDR homology-directed repair.

Back to article page