NAM-A-HOG Talk Series: Precision Genome Editing: Methods, Models, and Medicine

calendars.uark.edu

Abstract: Genome editing technologies have transformed our ability to interrogate gene function, model disease, and develop novel therapeutic strategies. While genome editing technologies are conceptually straightforward, the practical execution of precise genome engineering and model generation often requires extensive optimization, screening, and validation. I will discuss our efforts to expand access to genome editing technologies through the Center for Advanced Genome Engineering at St. Jude Children's Research Hospital. As a shared resource, the center provides expertise, infrastructure, and standardized workflows that enable investigators to efficiently implement established and emerging genome engineering approaches. Through these efforts, we have validated thousands of custom CRISPR reagents and supported the generation of over 1500 custom engineered cell lines, 400 preclinical animal models, and 150 pooled CRISPR screens to support a diverse research programs. I will also describe our work in developing innovative genome engineering methodologies that improve editing efficiency, outcomes, and scalability. By leveraging targeted deep sequencing and systematic analysis of editing outcomes across thousands of projects, we are generating data-driven approaches for CRISPR reagent design and optimization. Finally, I will discuss our efforts to advance genome editing therapies for hemoglobinopathies like sickle cell disease. Together, these efforts illustrate how methodological innovation, shared infrastructure, and translational research can work synergistically to democratize genome editing and accelerate the development of next-generation genetic medicines. About Shondra Pruett-Miller, Ph.D. Shondra Pruett-Miller, Ph.D., is an Associate Member in the Department of Cell and Molecular Biology at St. Jude Children's Research Hospital, where she also serves as Founding Director of the Center for Advanced Genome Engineering and Vice President of Shared Resource