Genome editing, also called gene editing and genomic engineering, is both a research tool and a therapeutic approach based on modifying genes. Gene editing research advances the understanding of genetic mechanisms and aids the development of therapies to treat genetic or acquired diseases. Genome-editing methods such as CRISPR-Cas9 are being deployed to study and treat cancer and sickle cell disease. St. Jude scientists are creating tools to understand better how the system works.
To schedule an interview with one of our experts, email media@stjude.org.
Genome Editing Experts
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Shondra Pruett-Miller, PhD
Pruett-Miller
Shondra Pruett-Miller, PhD, is the Center for Advanced Genome Engineering director and a faculty assistant member of Cell and Molecular Biology. Her research focuses on identifying, validating and delivering new genome editing applications and technologies, such as CRISPR/Cas9. Pruett-Miller can provide information associated with genetic predispositions associated with certain childhood cancers, stem cell research and current immunotherapy initiatives to create more effective treatments catered directly to each child.
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Akshay Sharma, MBBS
Sharma
Akshay Sharma, MD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, coordinates the bone marrow transplantation program for patients with sickle cell disease. Sharma is leading clinical research using gene editing, sometimes referred to as CRISPR therapeutics, for treating sickle cell disease. Gene editing for treating human disease is pushing the boundaries of science in healthcare. Sharma is also interested in the development of novel reduced-intensity and non-myeloablative conditioning regimens to improve sickle cell care.
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Shengdar Tsai, PhD, St. Jude Department of Hematology, is an expert in genome engineering, specifically protein engineering of CRISPR-Cas nucleases for precise gene correction. He recently developed CHANGE-seq, a high-throughput method for defining the genome-wide activity of genome editors. His work aids in developing genome editing approaches for the treatment of disease by ensuring that so-called “off-target” effects do not interfere with the therapy or create unwanted side effects.
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Mitchell Weiss, MD, PhD, St. Jude Department of Hematology chair, is a leading researcher on the biology of blood cell formation, non-malignant blood diseases and their treatments. His research interests include gene expression mechanisms during blood cell development, non-coding RNAs in red blood cell formation and hemoglobin structure and biology. At St Jude, Weiss has been investigating gene editing approaches for treating sickle cell disease.
