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Research Spotlight Webinar: "Developing regulated CRISPR for Huntington’s disease" with Beverly Davidson and Alejandro Mas Monteys, Children’s Hospital of Philadelphia, University of Pennsylvania

  • Hereditary Disease Foundation 601 W168th Street, Suite 54 New York, NY, 10032 United States (map)

About the Speakers

Beverly L. Davidson, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania

2015 HDF’s Leslie Gehry Brenner Prize for Innovation in Science Recipient

Dr. Davidson’s laboratory focuses on genetic diseases that affect the brain, investigating how mutant gene products contribute to disease, and why certain brain regions are more susceptible than others. Her team employs advanced molecular methods, sequencing and imaging in animal models, and a variety of molecular tools to test various hypotheses. Her lab is also developing next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments.

Alejandro Mas Monteys, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania

Dr. Mas Monteys is developing and testing a treatment for Huntington's disease using a novel allele specific CRISPR/Cas9 gene editing approach to eliminate expression of the abnormal protein causing HD. This technique offers an exciting extension of gene editing applications for HD and has significant implications for other human diseases.

The focus of Dr. Mas Monteys’s research and his unique expertise will continue to advance our understanding of the mechanisms of gene regulation by providing tools and technological methods to researchers across the world.

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