MedicalResearch.com Interview with:
Niren Murthy PhD
Professor of Bioengineering
University of California at Berkeley
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: In this paper we delivered Cas9 RNP in the brain using a delivery vehicle termed CRISPR-Gold. We were able to knock out the mGluR5 gene and rescue mice from autism using CRISPR-Gold. The background here is that there is a great need for safe and effective CRISPR delivery vehicles, and that brain gene editing has great therapeutic potential. This paper demonstrates for the first time that non-viral delivery of Cas9 RNP into the brain can have therapeutic effects.
MedicalResearch.com: What should clinicians and patients take away from your report?
Response: Brain gene editing has tremendous therapeutic potential, and can be achieved with non-viral Cas9 RNP delivery
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: We need to be able to edit the brains of large animals. The particles will need to be modified for this, we are currently working on this. GenEdit, a start-up company spun out from our lab, is also working on this.
Disclosures: I was a co-founder of GenEdit, but now have no equity in GenEdit, there should be no conflict of interest
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Bumwhee Lee, Kunwoo Lee, Shree Panda, Rodrigo Gonzales-Rojas, Anthony Chong, Vladislav Bugay, Hyo Min Park, Robert Brenner, Niren Murthy, Hye Young Lee. Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours. Nature Biomedical Engineering, 2018; DOI: 10.1038/s41551-018-0252-8
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