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Editorial

Unraveling the potential of CRISPR-Cas9 for gene therapy

, BS MS MS MBA PhD & , BA MA DPhil

Figures & data

Figure 1. Cas9-mediated DNA cleavage and genome editing. The Cas9 endonuclease forms a ribonucleoprotein complex in combination with the single guide RNA and the target dsDNA (top left). The Cas9:sgRNA complex first binds to PAM and drives the formation of an R-loop in the target DNA for genesis of a double-stranded break using the RuvC and HNH nickase domains (bottom left). The break may be repaired using either NHEJ (top right) or HDR (bottom right), to yield random and engineered genome modifications, respectively.

Figure 1. Cas9-mediated DNA cleavage and genome editing. The Cas9 endonuclease forms a ribonucleoprotein complex in combination with the single guide RNA and the target dsDNA (top left). The Cas9:sgRNA complex first binds to PAM and drives the formation of an R-loop in the target DNA for genesis of a double-stranded break using the RuvC and HNH nickase domains (bottom left). The break may be repaired using either NHEJ (top right) or HDR (bottom right), to yield random and engineered genome modifications, respectively.

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