September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
Getting the delivery right: Viral mediated applications of CRISPR in vivo
Author Affiliations & Notes
  • F Ann Ran
    Broad Institute of Harvard, Cambridge, Massachusetts, United States
    Society of Fellows, Harvard University, Cambridge, Massachusetts, United States
  • Footnotes
    Commercial Relationships   F Ann Ran, None
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science September 2016, Vol.57, No Pagination Specified. doi:
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      F Ann Ran; Getting the delivery right: Viral mediated applications of CRISPR in vivo. Invest. Ophthalmol. Vis. Sci. 201657(12):.

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      © 2017 Association for Research in Vision and Ophthalmology.

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Abstract

Presentation Description : The RNA-guided Cas9 nuclease from the bacterial CRISPR/Cas system has been adapted as a powerful tool for facilitating targeted genome editing in eukaryotes. Recently, we have identified an additional small Cas9 nuclease from Staphylococcus aureus that can be packaged with its guide RNA into a single adeno-associated virus (AAV) vector for in vivo applications. We demonstrate the use of this system for effective gene modification in adult animals and further expand the Cas9 toolbox for in vivo genome editing.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

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