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GEN’s editorial staff interviews life science academic and biotech industry leaders on important research, technology, and trends. These podcasts will keep you informed with all the important details you need.
Gene therapy to replace the faulty CLN2 gene, which causes a neurodegenerative disease that is fatal by age 8-12 years, was able to slow significantly the rate of neurologic decline in treated children, according to a paper published online ahead of print in the May 2008 issue (Vol. 19 No. 5) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The paper, entitled "Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis with CNS Administration of a Serotype 2 Adeno-associated virus expressing the CLN2 cDNA", is available free online at www.liebertpub.com/hum
During this week's GEN podcast, Dr. Ronald Crystal, one of the authors of the paper, addresses the key results of the research team's study. He provides details on the nature of Late Infantile Neuronal Ceroid Lipofuscinosis, also known as LINCL, including its causes, symptoms, and prevalence. Dr. Crystal also explains why the scientists chose the AAV gene therapy approach and talks about the benefits and side effects observed during the study. In addition, Dr. Crystal discusses both the challenges that remain for successful gene therapy for LINCL and his team's plans for future research in this area.
Ronald G. Crystal, MD is Professor and Chairman of the Department of Genetic Medicine of the Weill Medical College of Cornell University, where he is also the Bruce Webster Professor of Internal Medicine, Director of the Belfer Gene Therapy Core Facility and Chief of the Division of Pulmonary and Critical Care Medicine at the Weill Cornell-New York Presbyterian Hospital.
A pioneer in the field gene therapy, he was the first to use a recombinant virus as a vehicle for in vivo gene therapy, and has carried out human trials of gene therapy for cystic fibrosis, cardiac ischemia, cancer and central nervous system disorders. His current interests are deciphering how human genetic variation modulates gene expression in the context of environmental exposure and exploiting these relationships to recategorize human disease at the biologic level and identify who is at risk for disease.
In recognition of his significant accomplishments as a basic and clinical investigator, Dr. Crystal has received numerous professional honors including an honorary degree from the Johann Wolfgang Goethe University in Germany and an Honorary Fellowship from the Royal Physicians of Ireland. He serves on the editorial boards of numerous biomedical journals. He has published over 700 scientific articles, edited several textbooks, and served on a number of advisory boards to government and industry. Dr. Crystal is responsible for numerous biomedical patents and was the founder of GenVec, a biomedical company focused on gene therapy applications.