Figure - PMC

Skip to main content

An official website of the United States government

Here's how you know

Here's how you know

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

View full-text article in PMC

. 2022 Apr 12;13:1963. doi: 10.1038/s41467-022-29550-8

Search in PMC
Search in PubMed
View in NLM Catalog
Add to search

© The Author(s) 2022

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

PMC Copyright notice

Fig. 1 — AAV-delivered SpCas9 targets both the endogenous locus of a targeted gene and the inverted gRNA target sites flanking the donor DNA. Non-homologous end joining (NHEJ) recruits the donor DNA at the targeted locus. STOP codons knockout the endogenous gene while START sites initiate translation of the therapeutic gene. AAV adeno-associated viral vector, SpCas9 Streptococcus pyogenes Cas9, ITR inverted terminal repeats, U6-gRNA gRNA expression cassette containing the U6 promoter, the gene-specific or scramble gRNA and the gRNA scaffold; black rectangles and white triangles represent the two parts of the SpCas9 target sites flanking the SpCas9-induced double-strand break; scissors represent SpCas9-mediated double-strand breaks; STOP Stop codons in the 3 open reading frames, START translation start site (either kozak or internal ribosomal entry site, IRES), pA bovine growth hormone polyadenylation sequence; crossed scissors represent inability of SpCas9 to recognize target sites after homology-independent targeted integration has occurred.