Gene Therapy and Liver Transplants for a Birth Defect
Mark L. Batshaw, M.D.
Developmental Pediatrics
Senior Investigator, Center for Genetic Medicine Research
Children's National Hospital
Professor of Pediatrics
The George Washington University
School of Medicine and Health Sciences
Maury Strauss Distinguished Public Lecture: Gene Therapy and Liver Transplants for a Birth Defect
Date: November 4, 2021
Time: 5:30 - 6:30 p.m.
Archived video
About this Seminar
Urea cycle disorders are a group of rare genetic metabolic disorders that are associated with episodes of coma and brain damage. This is due to an accumulation of ammonia that is normally detoxified in the liver through a cycle of enzymes that, when functioning normally, leads to the excretion of toxic waste from protein metabolism as non-toxic urea. While urea cycle disorders occur in only about 1 in 14,000 people, rare genetic disorders as a whole occur in about 1 in 15 people. Using urea cycle disorders as a model of rare genetic disease research, in this lecture the history of the development of therapies for these disorders, the successes and failure, is discussed by Dr. Batshaw, an investigator who has worked in this field for 45 years. The current outcomes are discussed from a neurodevelopmental perspective. A major focus will be on the potential use of gene therapy and gene editing to cure these disorders.
Additional Details
This is a free event hosted by Dr. Michael Friedlander and the Fralin Biomedical Research Institute. For more information, please call 540-526-2059.
About Maury Strauss
Maury Strauss is a longtime community benefactor and businessman who supports biomedical research with the goal of energizing the local economy and improving quality of life in our neighborhoods and around the world. In order to ensure the continued success of Roanoke’s biomedical research enterprise, as well as the free public seminars, Mr. Strauss has made a generous gift to the series. Read VT News Story
Support Our Research
Your generous support of the Fralin Biomedical Research Institute's rigorous biomedical research enterprise makes a difference for our faculty, students, and patients. Every donation helps accelerate the pace of new discoveries to help patients with cancer, neurological disorders, heart disease, and even rare genetic disorders. Private donations fast-track our progress.
You May Also Be Interested In...
-
Home Item
Molecular and Neurobiological Studies in Rett Syndrome and Other MECP2 Disorders , homeMay 1, 2025, 5:30 p.m. (Reception at 5 p.m.) | Huda Zoghbi, M.D., Professor, Pediatrics, Molecular and Human Genetics, Neuroscience, and Neurology; Director, Jan and Dan Duncan Neurological Research Institute, Baylor College of Medicine; Member, National Academy of Medicine | Maury Strauss Distinguished Public Lecture
-
Home Item
A Novel Bispecific Antibody to Target the Brain Tumor Microenvironment , homeMay 2, 2025, 11:00 a.m. | Khuson Yun, Ph.D., Associate Professor, Department of Neurology, Houston Methodist Chair, Neurodegenerative Disease Research, Houston Methodist Research Institute, Weill Cornell Medical College | Co-Sponsored by the Fralin Biomedical Research Institute Cancer Research Center – Roanoke
-
Home Item
Transformation of Information from the External World by Cortical Circuits During Learning , homeMay 9, 2025, 11:00 a.m. | Alison Barth, Ph.D., Professor, Department of Biological Sciences, Carnegie Mellon University | Co-Sponsored by the Center for Neurobiology Research, Fralin Biomedical Research Institute
-
Home Item
The Role of Exercise in Memory Function , homeMay 23, 2025, 11:00 a.m. | Henriette van Praag, Ph.D., Associate Professor, Department of Biomedical Science, Charles E. Schmidt College of Medicine, Florida Atlantic University | Co-Sponsored by the Center for Exercise Medicine Research, Fralin Biomedical Research Institute
-
Home Item
Pathways and Regulation of Cardiac Nav1.5 Channel Trafficking in Adult Myocytes , homeMay 30, 2025, 11:00 a.m. | Gea-Ny Tseng, Ph.D., Professor, Department of Physiology and Biophysics, Virginia Commonwealth University School of Medicine | Co-Sponsored by the Fralin Biomedical Research Institute Center for Vascular and Heart Research
-
Home Item
Targeting Acquired Dependencies During Tumor Evolution , homeJune 6, 2025, 11:00 a.m. | Kris Wood, Ph.D., Associate Professor, Department of Pharmacology and Cancer Biology, Duke University School of Medicine | Co-Sponsored by the Fralin Biomedical Research Institute Cancer Research Center – D.C.