Biomedicine Seminar

Abstract
The CRISPR/Cas gene editing platform has emerged as a powerful and Nobel prize-winning tool for making precise edits to the genome of living organisms. In this presentation, I will present some of our contributions to applying this technique to blood stem cells, and I will provide an overview of the current status of therapeutic gene editing to treat monogenic disorders of the blood. Finally, I will present some of our recent work on repurposing CRISPR/Cas for targeted transcriptional activation and interference (CRISPRa and CRISPRi).