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Seminar Series: “In vivo delivery of Cas9 ribonucleoprotein and donor DNA with gold nanoparticles” featuring Professor Niren Murthy, UC Berkeley

October 2, 2017 @ 11:00 am - 12:00 pm

Department Seminar Series:

In vivo delivery of Cas9 ribonucleoprotein and donor DNA with gold nanoparticles

Niren Murthy

Niren Murthy
Professor of Bioengineering
University of California, Berkeley

October 2, 2017
11:00 AM – 12:00 PM
Natural Sciences I, Room 1114

Abstract

Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases because of their ability to generate homologous DNA recombination (HDR) and correct DNA mutations.  However, viral gene therapy is currently the only delivery technology available for generating HDR in vivo with Cas9, and is challenging to bring into clinical trials because of off-target DNA damage and immunogenicity. In this presentation, I will describe a non-viral Cas9 delivery vehicle, termed CRISPR-Gold, which can induce HDR in vivo by directly delivering Cas9 protein, gRNA, and donor DNA.  CRISPR-Gold is composed of gold nanoparticles assembled with the Cas9/gRNA ribonucleoprotein (RNP) complex, donor DNA, and an endosomal disruptive polymer. We have been able to demonstrate that CRISPR-Gold can correct the DNA mutation that causes Duchenne muscular dystrophy (DMD) in mdx mice via HDR, with an efficiency of 5.4% after an intramuscular injection and with minimal levels off-target DNA damage. In addition, we demonstrate that CRISPR-Gold can improve muscle strength and lower tissue fibrosis in mdx mice. CRISPR-Gold is the first example of a non-viral delivery vehicle that can generate HDR in vivo and has tremendous potential for treating DMD and other genetic diseases caused by single base pair mutations.

About Professor Niren Murthy

Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley. Dr. Murthy’s laboratory is an interdisciplinary laboratory that focuses on the development of new materials for drug delivery and molecular imaging. Dr. Murthy received the NSF CAREER award in 2006, and the 2009 Society for Biomaterials Young Investigator Award.  The Murthy laboratory has developed several new biomaterials for drug delivery and molecular imaging, such as the hydrocyanines.

Event Sponsors

UCI Department of Pharmaceutical Sciences

UCI Department of Biomedical Engineering

Details

Date:
October 2, 2017
Time:
11:00 am - 12:00 pm
Event Tags:
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Venue

Natural Sciences I, Room 1114
Natural Sciences I, Room 1114
Irvine, CA 92697 United States
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