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Gene Therapy and Liver Transplants for a Birth Defect

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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. 

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