Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be developed before the therapeutic potential of CRISPR based therapeutics can be realized. In this presentation, Dr. Niren Murthy will describe a non-viral Cas9 delivery vehicle, termed CRISPR-Gold, which can induce homology directed DNA repair (HDR) in vivo by directly delivering Cas9 protein, gRNA, and donor DNA.
From this webinar, you will learn the following:
- Non-viral delivery of the Cas9 protein possible in vivo
- Gene editing after a local injection can have therapeutic effects
- New strategies for reversibly modifying proteins.
Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley. Dr. Murthy’s scientific career has focused on the molecular design and synthesis of new materials for drug delivery and molecular imaging. The Murthy laboratory developed the hydrocyanines in 2009, which are now one of the most commonly used probes for imaging reactive oxygen species and commercially available from multiple sources. The Murthy laboratory has developed several new nanoparticulate technologies for drug delivery, such as the polyketals, which have been used by numerous laboratories to enhance the delivery of small molecules and proteins. The Murthy laboratory has been recently focused on developing non-viral delivery vehicles that can deliver Cas9 protein, gRNA and Donor DNA in vivo. Dr. Murthy received the NSF CAREER award in 2006, and the 2009 Society for Biomaterials Young Investigator Award.