Discovery and Innovation Speaker Series: “Correction of Duchenne Muscular Dystrophy by Genome Editing”
Presented by:
Eric N. Olson, PhD
Annie and Willie Nelson Professor in Stem Cell Research
Pogue Distinguished Chair in Research on Cardiac Birth Defects
The Robert A. Welch Distinguished Chair in Science
The University of Texas Southwestern Medical Center
Eric Olson, PhD is the founding chair of the Department of Molecular Biology at UT Southwestern Medical Center. Dr. Olson and his trainees discovered many of the genes that control heart and muscle development and disease, revealing fundamental principles of organ formation and providing new concepts in the quest for cardiovascular therapeutics. Dr. Olson is a member of the U.S. National Academy of Sciences, the National Academy of Medicine, and the American Academy of Arts and Sciences. His work has been recognized by numerous awards, including the Passano Award and the March of Dimes Prize in Developmental Biology.
Talk Summary:
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by mutations in the Dystrophin gene, which encodes a large intracellular protein that maintains integrity of muscle cell membranes. More than 4,000 DMD mutations have been identified in humans. The majority of mutations are deletions that cluster in hot spots, such that skipping of out-of-frame exons can potentially restore the reading frame of the Dystrophin protein. We have used CRISPR/Cas9 to generate new mouse models of DMD lacking the most prominently deleted Dystrophin exons in humans. To permanently correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, we have used adeno-associated virus-9 (AAV9) to deliver CRISPR/Cas9 gene editing components to dystrophic mice, a method we refer to as Myoediting. We have also optimized Myoediting of many types of DMD mutations in muscle cells derived from iPS cells generated from blood samples of DMD patients. In a path toward clinical translation of gene editing for DMD, current efforts are directed toward correction of DMD mutations in large mammals. Opportunities and challenges in the path toward permanent correction of disease-causing mutations responsible for DMD and other monogenic disorders by genomic editing will be discussed.
Hosted by:
Associate Professor
Department of Surgery, Biochemistry and Molecular Medicine
Saban Research Institute of Children’s Hospital Los Angeles
Keck School of Medicine of University of Southern California
More Information:
Lunch will be provided to seminar guests; first come, first served
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