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Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies

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Abstract

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder in which the loss of dystrophin causes progressive degeneration of skeletal and cardiac muscle. Potential therapies that carry substantial risk, such as gene- and cell-based approaches, must first be tested in animal models, notably the mdx mouse and several dystrophin-deficient breeds of dogs, including golden retriever muscular dystrophy (GRMD). Affected dogs have a more severe phenotype, in keeping with that of DMD, so may better predict disease pathogenesis and treatment efficacy. Various phenotypic tests have been developed to characterize disease progression in the GRMD model. These biomarkers range from measures of strength and joint contractures to magnetic resonance imaging. Some of these tests are routinely used in clinical veterinary practice, while others require specialized equipment and expertise. By comparing serial measurements from treated and untreated groups, one can document improvement or delayed progression of disease. Potential treatments for DMD may be broadly categorized as molecular, cellular, or pharmacologic. The GRMD model has increasingly been used to assess efficacy of a range of these therapies. A number of these studies have provided largely general proof-of-concept for the treatment under study. Others have demonstrated efficacy using the biomarkers discussed. Importantly, just as symptoms in DMD vary among patients, GRMD dogs display remarkable phenotypic variation. Though confounding statistical analysis in preclinical trials, this variation offers insight regarding the role that modifier genes play in disease pathogenesis. By correlating functional and mRNA profiling results, gene targets for therapy development can be identified.

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Acknowledgments

These studies were supported by the following grants: AAV-Mediated Gene Therapy in Canine Muscular Dystrophy, Project 2, University of Pittsburgh Wellstone MDCRC (1U54AR50733; NIAMS) (Xiao); Gene Therapy in Golden Retriever Muscular Dystrophy Model, Project 2, University of North Carolina-Chapel Hill Wellstone MDCRC (5U54AR056953; NIAMS) (Xiao); the Co-operative Program in Translational Research: Proposal for Establishment of the National Center for Canine Models of Duchenne Muscular Dystrophy (NCDMD) (1U24NS059696-01A1; NINDS) (Kornegay); Natural History and Immunological Parameters in the German Shorthaired Pointer Muscular Dystrophy (GSHPMD) Dog, Translational Research Advisory Committee (TRAC) of the Muscular Dystrophy Association (Kornegay); F32 NRSA grant 1F32AR060703-01 (NIAMS) (Nghiem); and the National Center for Medical Rehabilitation Research Core (5R24HD050846; NCMRR) (Hoffman).

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  1. Ratna K. Bhavaraju-Sanka

    Present address: Department of Neurology, University of Texas Health Sciences Center, Medical Arts and Research Center (MARC), 8300 Floyd Curl Drive, MSC 7883, San Antonio, TX, 78229-3900, USA

Authors and Affiliations

  1. Department of Pathology and Laboratory Medicine, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan & David A. Detwiler

  2. Department of Neurology, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Joe N. Kornegay, Ratna K. Bhavaraju-Sanka, James F. Howard Jr. & Zheng Fan

  3. The Gene Therapy Center, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan & Xiao Xiao

  4. School of Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina-Chapel Hill, Campus Box 7525, Chapel Hill, NC, 27599, USA

    Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Juan Li, Ratna K. Bhavaraju-Sanka, James F. Howard Jr., Zheng Fan & Xiao Xiao

  5. Eshelman School of Pharmacy, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Juan Li & Xiao Xiao

  6. Department of Neurology, Institute for Regenerative Medicine, Wake Forest University, Winston-Salem, NC, 27157, USA

    Martin K. Childers

  7. Department of Integrative Systems Biology, George Washington University School of Medicine, and Research Center for Genetic Medicine, Children’s National Medical Center, 111 Michigan Ave. NW, Washington, DC, 20010, USA

    Peter Nghiem & Eric P. Hoffman

  8. Department of Human Genetics, University of Utah, 15 North 2030 East, Salt Lake City, UT, 84112, USA

    C. Aaron Larsen

  9. Department of Human Nutrition, Foods, and Exercise, Virginia Tech University, 321 Wallace Hall, Blacksburg, VA, 24061, USA

    Robert W. Grange

  10. Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, 1052 William Moore Drive, Raleigh, NC, 27607, USA

    Sandra Tou & Bruce P. Keene

  11. Department of Psychiatry, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Jiahui Wang

  12. Veterinary Emergency and Specialty Center of Santa Fe, 2001 Vivigen Way, Santa Fe, NM, 87505, USA

    Scott J. Schatzberg

  13. Departments of Computer Science and Psychiatry, University of North Carolina-Chapel Hill, Chapel Hill, NC, 27599, USA

    Martin A. Styner

  14. Nationwide Children’s Hospital, Center for Gene Therapy, WA4023, 700 Children’s Drive, Columbus, OH, 43209, USA

    Kevin M. Flanigan

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Correspondence to Joe N. Kornegay.

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Kornegay, J.N., Bogan, J.R., Bogan, D.J. et al. Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies. Mamm Genome 23, 85–108 (2012). https://doi.org/10.1007/s00335-011-9382-y

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