39
EVENT FORMAT
Lecture
2020
Januar y
12-2 3
Genome Editing Based Approaches for Personalized Medicine
DESCRIPTION
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.
SPEAKER Gang Bao
CREDITS 1
Foyt Family Chair Professor in the Department of Bioengineering and Associate
Dean for Research and Innovation at Rice University
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.