The Department of Biochemistry & Molecular Genetics presents:
Gregory Newby, PhD
Assistant Professor of Genetic Medicine
Johns Hopkins
Presentation:
"Base Editing and Prime Editing to Address Genetic Disease"
Abstract:
Our recent work has demonstrated that it is possible to treat mouse models of genetic disease using LNPs, AAVs, and virus-like particles to access and edit the liver, heart, brain, and vasculature of mice. Ex vivo modification of hematopoietic stem cells has permitted treatment of sickle cell mouse models and we are advancing towards in vivo delivery to the blood. Early successes in the clinic are motivating expansion of in vivo editing approaches for rare disease. My lab is collaborating with the JHU Hemophilia Treatment Center to develop personalized treatments for rare forms of hemophilia. This seminar will overview these recent demonstrations and ongoing efforts.
Host: Dr. Marc Mendillo, PhD
Refreshments will be served.