Center of Research Translation in Muscular Dystrophy Therapeutic Development :: Nationwide Children's Hospital

Center of Research Translation (CORT) in Muscular Dystrophy Therapeutic Development

Mission and Objectives

Based at the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital, the Center of Research Translation (CORT) in Muscular Dystrophy Therapeutic Development makes use of the experience of institutional investigators in bringing AAV-based and other genetic therapies through preclinical development to investigator-sponsored and industry-partnered clinical trials, concentrating on novel approaches to Duchenne, limb-girdle, congenital, and facioscapulohumeral muscular dystrophies.

The overall objective of the CORT is to enhance the rapid translation of new basic science discoveries into gene therapies for muscular dystrophies. The CORT is consistent with the mission of the National Institutes of Health's National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), which has the goal of finding effective treatments for and improving the quality of life of patients with debilitating forms of muscle disease.

>> Learn more about the NIH/NIAMS Centers of Research Translation

Center Goals

The Center for Gene Therapy has a dedicated translational program that targets muscular dystrophies, with a particular longstanding interest in developing meaningful therapies for the most common forms, including Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). 

Our Center's goals include unraveling disease pathogenesis and developing new treatment paradigms that can be translated from the bench to the bedside. All three projects below make use of two critical research cores: the Therapeutic Viral Vector Design and Development Research Core (PI: Louise Rodino-Klapac, PhD) and the Muscular Dystrophy Cell and Serum Banking Core (PI: Kim McBride, MD).

Project 1: Principal Investigator - Paul Martin, PhD

Project 1 seeks to extend a therapy now entering trials in DMD to other forms of muscular dystrophy, by applying the overexpression of Galgt2, an enzyme that alters skeletal muscle glycosylation to boost the expression of proteins that amerliorate disease.

>> Learn more about the Martin Lab in the Center for Gene Therapy

Project 2: Principal Investigator - Scott Harper, PhD

Project 2 explores novel approaches to modulating the expression of the DUX4 gene to treat the relatively common and debilitating FSHD.

>> Learn more about the Harper Lab in the Center for Gene Therapy

Project 3: Principal Investigator - Kevin Flanigan, MD

Project 3 seeks to rapidly translate a newly discovered mechanism for dystrophin translational control into meaningful therapy for boys with DMD.

>> Learn more about the Flanigan Lab in the Center for Gene Therapy