Where: Building 9 Lecture hall 1
Credit: 1
Description
Lecture by Gang Bao, Foyt Family Chair Professor in the Department of Bioengineering and Associate Dean for Research and Innovation at Rice University.
ABOUT THIS EVENT
With the recent advent in genome editing using engineered nucleases such as CRISPR/Cas9 systems, we are in a new era of personalized medicine, and many diseases can be treated or cured based on the individual’s genome. However, significant challenges exist to treat a disease with high efficacy and safety using genome editing based approaches. In this talk, Professor Bao will first focus on the development of a CRISPR/Cas9 based genome editing approach to treat sickle cell disease (SCD), which is a monogenic disorder that affects millions worldwide. Professor Bao and his team have systematically optimized the gRNA and ssODN template designs, achieved high HBB gene editing rates in human CD34+ HSPCs derived from the peripheral blood and bone marrow of patients with SCD. They have also performed a genome-wide unbiased analysis of off-target effects and demonstrated that gene-edited CD34+ HSPCs derived from SCD patients were able to engraft into the bone marrow of NSG mice, with the corrected alleles stable for up to 19 weeks post-transplantation. He will also illustrate the need to perform off-target analysis based on the individual’s genome, and the design of corrective donor templates according to specific gene defects that cause disease. Finally, he will discuss the challenges of in vivo genome editing and describe a unique delivery vehicle that combines magnetic nanoparticles (MNP) and baculoviral (BV) vector for CRISPR/Cas9 based in vivo genome editing with spatial control.
The Academic Lectures are a series of lectures featuring a range of in-Kingdom and international entrepreneurs, academics and distinguished guest speakers.
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Gang Bao
Gang Bao is the Foyt Family Chair Professor in the Department of Bioengineering, and Associate Dean for Research and Innovation of School of Engineering, Rice University. He is also the Director of the Nanomedicine Center for Nucleoprotein Machines at Rice. He is a Fellow of the American Association of Advancement in Science, American Society of Mechanical Engineers, American Physical Society, American Institute for Medical and Biological Engineering, and Biomedical Engineering Society. His research focuses on the development of nanotechnology and biomolecular engineering tools for biological and disease studies, including multifunctional magnetic nanoparticles and engineered nucleases such as CRISPR/Cas9. These approaches have been applied to the diagnosis and treatment of chronic diseases such as cancer, and genome editing approaches for treating single-gene disorders.
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