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

About this event

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 Resources. A proud University of Arkansas alumna, she earned her B.S. while conducting honors thesis research as a SURF Scholar in the laboratory of Inés Pinto. She went on to earn her Ph.D. at The University of Texas Southwestern Medical Center, where she trained in Matthew Porteus's laboratory and helped advance the development of zinc finger nucleases for genome editing applications.

With more than two decades of experience in genome engineering across both industry and academia, Dr. Pruett-Miller has played a key role in the development and implementation of genome editing technologies. Before joining St. Jude in 2017, she held positions at Sigma-Aldrich and Washington University School of Medicine. Through the Center for Advanced Genome Engineering, she has supported the creation of more than 1,500 engineered cell lines, over 400 preclinical animal models, and more than 150 pooled CRISPR screens, enabling discoveries in basic biology and disease research. Her current research focuses on expanding access to genome editing technologies, developing innovative genome engineering methods, and advancing genome editing therapies for hemoglobinopathies and other genetic diseases.