- Graduated medical school from Rush Medical College
- Completed residency at Johns Hopkins Hospital
- Completed fellowship at Johns Hopkins Hospital and University of Utah Medical School
- Director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital
- Current research includes a wide range of projects ranging from clinical datasets to basic science projects exploring gene delivery and gene correction for various forms of muscular dystrophy; the specific project will depend upon the trainee and the amount of time they will be able to commit
- Research area: Neuromuscular, Gene therapy
- Clinical and basic science research projects available
- Open to mentoring: current neurology resident or fellow, current neurology pre-lim, medical students
From Dr. Flanigan: “I am the director of the Nationwide Children’s Hospital (NCH) Center for Gene Therapy, as well as director of the NCH Neuromuscular Program and its neuromuscular and multidisciplinary Muscular Dystrophy clinics. I am also director of the NCH NIAMS P50-funded Center of Research Translation in Muscular Dystrophy Therapeutics.
I trained in Neurology (residency) and Neuromuscular Disease (fellowship) at the Johns Hopkins Hospital before doing a laboratory-based fellowship in Molecular Biology and Genetics at the University of Utah, where I joined the faculty prior to moving to Columbus in 2009.
I founded and have since 2012 directed the NCH/OSU Myology Course, direct the OSU/NCH Neuromuscular Fellowship program pediatric component, and established the NCH GME-approved Neuromuscular Genetic Therapeutics (now Gene Therapy) fellowship. My work is primarily funded by the NIH, with additional biopharma partner sponsored research agreements and foundation funding.
The overall mission of my laboratory is to rapidly advance translational therapies for neuromuscular and neurologic diseases. Our particular emphasis is on the muscular dystrophies and congenital myopathies.
Our laboratory is concentrating on two major overarching themes.
The first is understanding molecular mechanisms by which patients with muscular dystrophy – why, for example, certain patients who are expected to make no dystrophin and have severe Duchenne muscular dystrophy instead have mild Becker muscular dystrophy. We are cataloging clinical features, protein expression studies from muscle biopsies using cutting-edge microscopy and capillary immunoassay techniques, and RNA Sequencing studies.
The second is to sidestep the limitation imposed by adeno-associated viruses (AAVs) regarding gene length that require delivery of engineered microdystrophin constructs, and to leapfrog to methods for expressing full-length, wild-type dystrophin. We use a variety of techniques, include CRISPR/Cas gene editing, and U7snRNA pre-mRNA splice alteration (the latter of which is already in clinical trial).
The range of our work is found here: https://www.ncbi.nlm.nih.gov/myncbi/k.flanigan.1/bibliography/public/
There are no pre-requisites. The level of independence for a given project will depend on prior experiences.
There are no time frame restrictions. Any trainee is expected to pre-define a work schedule with immediate mentors (which may include lab technicians, clinical research coordinators, graduate student, or post-doctoral trainees) and within reason will be expected to commit to that schedule in order to maximize their meaningful exposure to research.”