MDX Mouse as Therapeutic Model System: Development and Implementation of Phenotypic Monitoring
As Michael Brooke so aptly stated at the beginning of this session on phenotypic monitoring, it is imperative to have a quantifiable test system in place prior to assessing treatment entities for any of the muscular dystrophies (Brooke et al., 1981). In this manner, the various medical, experimental, and ethical considerations have been resolved in advance providing an unequivocal foundation for determining efficacy (regardless of the particular therapeutic approach under consideration). Similarly, in the preclinical study of myopathic animal models, it is equally pertinent to establish reliable phenotypic endpoints before the implementation of a therapeutic study. Hence, efficacy or a lack thereof can be objectively and rationally determined against a backdrop of standardized markers of the disease.
KeywordsMuscular Dystrophy Duchenne Dystrophy Spinal Muscular Atrophy Duchenne Muscular Dystrophy Activity Chamber
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- Cooper, B. J., Winand, M. J., Stedman, H., Valentine, B. A., Hoffman, E. P., Kunkel, L, M., Scott, M. O., Fishbeck, K. H., Kornegay, J. N., Avery, R. J., Williams, J. R., Schmechel, R. D. and Sylvester, J. E., 1988, The homologue of the duchenne locus is defective in X-linked muscular dystrophy of dogs, Nature, 334: 154–156.Google Scholar
- Hudecki, M. S., Kibler, P. K., Davis, P. J., Davis, F. B., Thacore, H. R., Pollina, C. M. and Blas, S. D., 1986, Abnormal gene expression of calmodulin in dystrophic chicken muscle, Res. Commun. Biochem. Biophys., 137: 507–512.Google Scholar
- Hudecki, M. S., Pollina, C. M., Bhargava, A. K., Hudecki, R. S., 1980, Screening of anti-seritoninergic drugs employing the genetically dystrophic chicken, Arch. Neurol., 73: 173–185.Google Scholar
- Hudecki, M. S., Pollina C. M., Heffner, R. R. and Bhargava, A. K., 1981, Enhanced functional ability in drug-treated dystrophic chickens: Trial results with indomethacin, diphenylhydantoin and prednisolone, Exp. Neurol., 73: 173-185.Google Scholar
- Karpati, G., Pouliot, Y., Carpenter, S. and Holland, P., 1989, Implantation of nondystrophic allogenic myoblasts into dystrophic muscles of mdx mice produces “mosaic” fibers of normal microscopic phenotype, in: “Cellular and Molecular Biology of Muscle Development”, Alan R. Liss, Inc., New York.Google Scholar
- Law, P. K. and Schafer, B., 1989, Personal communication.Google Scholar
- Morgan, J. E. and Partridge, T. A., 1989, Personal communication.Google Scholar
- Partridge, T. A., Morgan, Coulton, G. R., Hoffman, E. P. and Kunkel, L. M., 1989, Conversion of mdx myofibers from dystrophin-negative to -positive by injection of normal myoblasts, Nature, 337: 176–179.Google Scholar
- Zapalowski, C. hudeck, M. S., Davis, F. B., Davis, P. J., Pollina, C. M. and Blass, S. D., 1989, Characterization of a biochemical abnormality in the murine model (mdx) of muscular dystrophy, Clin. Res., 37: 464a.Google Scholar