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. Author manuscript; available in PMC: 2020 Oct 6.
Published in final edited form as: Am J Med Genet C Semin Med Genet. 2016 Nov 8;172(4):349–366. doi: 10.1002/ajmg.c.31534

Figure 6. CRISPR/Cas9 delivery.

Figure 6.

Illustration depicting how CRISPR/Cas9 can be packaged for gene-modifying therapy. Cas9 and gRNA are delivered via a vector without donor template (left) or with donor template (right). When given without donor template, gene ablation can be achieved at a target sequence specified by gRNA. Cas9 variants can be used to create a shorter sequenced, single construct. Precise homology-directed repair is achieved when the vector includes a donor template. ITR sequences allow identification and encapsulation of the construct by the viral vector. ITR, inverted terminal repeat; gRNA, guide RNA; Cas9, CRISPR-associated protein 9.