32 episodios

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.

This Month in Muscular Dystrophy Nationwide Children's Hospital

    • Ciencias

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.

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

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

    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.

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

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

    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.

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

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

    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.

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

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

    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.

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

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

    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.

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

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

    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).

    • 10 min

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