Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comment
. 2016 Mar;24(3):414-6.
doi: 10.1038/mt.2016.29.

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

Thierry VandenDriessche  1   2 , Marinee K Chuah  1   2
Affiliations
Comment

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

Thierry VandenDriessche et al. Mol Ther. 2016 Mar.
No abstract available

PubMed Disclaimer

Figures

Figure 1
Figure 1
In vivo somatic muscle-directed editing with CRISPR/Cas9. The Cas9 and guide RNA (gRNA) expression cassettes were packaged into an adeno-associated virus (AAV) vector that exhibits improved myotropic and cardiotropic properties (AAV8 or AAV9). The Staphylococcus aureus or S. pyrogenes Cas9 was expressed from a cytomegalovirus (CMV) promoter (or a smaller derivative), whereas the gRNAs were expressed from the U6 pol III promoter. The gRNAs were specifically designed to target the defective exon 23 of the endogenous mouse dystrophin gene of the mdx mouse, which contains a nonsense mutation resulting in a defective dystrophin protein. The vectors were injected either systemically or locally into the muscle in adult or newborn mdx mice. NHEJ, nonhomologous end joining.
See this image and copyright information in PMC

Comment on

References

    1. Ervasti, JM, Ohlendieck, K, Kahl, SD, Gaver, MG and Campbell, KP (1990). Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle. Nature 345: 315–319. - PubMed
    1. Campbell, KP and Kahl, SD (1989). Association of dystrophin and an integral membrane glycoprotein. Nature 338: 259–262. - PubMed
    1. Xu, L, Park, KH, Zhao, L, Xu, J, El Refaey, M, Gao, Y et al. (2016). CRISPR-mediated genome editing restores dystrophin expression and function in mdx mice. Mol Ther 24: 564–569. - PMC - PubMed
    1. Long, C, Amoasii, L, Mireault, AA, McAnally, JR, Li, H, Sanchez-Ortiz, E et al. (2016). Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351: 400–403. - PMC - PubMed
    1. Nelson, CE, Hakim, CH, Ousterout, DG, Thakore, PI, Moreb, EA, Castellanos Rivera, RM et al. (2016). In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351: 403–407. - PMC - PubMed