Guest: Jerry Mendell, MD, is Director of the Center for Gene Therapy in The Research Institute, Director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, and Curran-Peters Chair of Pediatric Research at Nationwide Children’s Hospital. He is also an attending neurologist at Nationwide Children’s and a professor of pediatrics and neurology at The Ohio State University College of Medicine. • View the published abstract for this month’s featured research article. • Learn more about the gene therapy trial for Becker Muscular Dystrophy.

This Month in Muscular Dystrophy

Dr. Jerry Mendell Discusses Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy :: March 2015

Guest Host: Scott Harper, PhD, is a principal investigator in the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital. Guest: Kevin Flanigan, MD, principal investigator in the Center for Gene Therapy, is also an attending neurologist at Nationwide Children’s and professor of Pediatrics and Neurology at The Ohio State University College of Medicine. View the published abstract for this month’s featured research article. Learn more about the Flanigan Lab and their research.

Dr. Kevin Flanigan Discusses IRES-Induced Dystrophin as a Potential Therapy for DMD

Guest: Louise Rodino-Klapac, PhD, is a principal investigator in the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital. She is also an assistant professor in the Department of Pediatrics at The Ohio State University College of Medicine. • View the published abstract for this month’s featured research article. • Learn more about Dr. Rodino-Klapac’s gene therapy research for muscular dystrophies.

Dr. Louise Rodino-Klapac Discusses Dysferlin Overlap Vectors to Restore Function in Dysferlinopathies

Guest: Scott Harper, PhD, is a principal investigator in the Center for Gene Therapy in The Research Institute and the Neuromuscular Disorders program at Nationwide Children’s Hospital. He is also a faculty member for the Child Neurology Residency program at Nationwide Children’s and an assistant professor in the Department of Pediatrics at The Ohio State University College of Medicine. • View the published abstract for this month’s featured research article. • Learn more about Dr. Harper’s research on neuromuscular disorders.

Dr. Scott Harper Discusses RNAi silencing in Limb Girdle Muscular Dystrophy 1A

Dr. Brian Kaspar Discusses a New Method for Studying ALS, with Potential to Test Therapeutics in Individual Patients :: April 2014 Guest: Brian Kaspar, PhD, is a principal investigator in the Center for Gene Therapy and the first recipient of the Grant Morrow, III, MD, Endowed Chair in Pediatric Research in The Research Institute at Nationwide Children’s Hospital. He is also an associate professor in the departments of Pediatrics and Neuroscience at The Ohio State University College of Medicine, and a recognized national expert in the discovery of new therapies for spinal muscular atrophy and amyotrophic lateral sclerosis.

Dr. Brian Kaspar Discusses a New Method for Studying ALS, with Potential to Test Therapeutics in Individual Patients

Dr. Louis Chicoine Discusses the Effect of Plasmapheresis in Removal of AAV Antibodies for Gene Therapy :: November 2013 Guest: Louis Chicoine, MD, principal investigator in the Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital; assistant professor of Pediatrics at The Ohio State University College of Medicine. Access an abstract of this month’s featured research article: Plasmapheresis eliminates the negative impact of AAV antibodies on micro-dystrophin gene expression following vascular delivery. Molecular Therapy. 2013 Oct 23. [Epub ahead of print] Major contributors to the work described include the Jesse’s Journey Foundation, the Nationwide Children’s Hospital Foundation, the Muscular Dystrophy Association, a National Institutes of Health grant (U54 NS055958), and a Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center grant (U54 HD066409).

Dr. Louis Chicoine Discusses the Effect of Plasmapheresis in Removal of AAV Antibodies for Gene Therapy

Dr. Volker Straub Discusses the Use of Muscle MRI in Muscular Dystrophy Guest: Volker Straub, MD, PhD, Harold Macmillan Professor of Medicine and Professor of neuromuscular genetics at the University of Newcastle upon Tyne in the United Kingdom. Access an abstract of this month’s featured research articles: - Quantitative Muscle MRI as an Assessment Tool for Monitoring Disease Progression in LGMD2I: A Multicentre Longitudinal Study. PLoS One. 2013 Aug 14, 8(8):e70993. - MR imaging in Duchenne muscular dystrophy: Longitudinal assessment of natural history over 18 months. Muscle Nerve. 2013 Apr 26.

Dr. Volker Straub Discusses the Use of Muscle MRI in Muscular Dystrophy

Dr. Jeff Chamberlain Discusses Gene and Cell Mediated Therapies for Muscular Dystrophy Guest: Jeff Chamberlain, PhD, McCaw Chair in Muscular Dystrophy and Professor in the departments of neurology, medicine and biochemistry at the University of Washington. Access an abstract of this month’s featured article: Gene and cell-mediated therapies for muscular dystrophy. Muscle Nerve. 2013 May, 47(5):649-63. Epub 2013 Mar 29.

Dr. Jeff Chamberlain Discusses Gene and Cell Mediated Therapies for Muscular Dystrophy

Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. During each podcast, authors of recent publications discuss how their work improves our understanding of inherited neuromuscular diseases, and what their work might mean for treatment of these diseases.

hubhopper

This Month in Muscular Dystrophy

hubhopper