Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Year: 2015
Type: review
Abstract: Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal models of DMD have been developed over the last 30 years, ranging from invertebrate to large mammalian models. mdx mice are the most commonly employed models in DMD research and have been used to lay the groundwork for DMD gene therapy. After ~30 years of development, the field has reached the stage at which the results in mdx mice can be validated and scaled-up in symptomatic large animals. The canine DMD (cDMD) model will be excellent for these studies. In this article, we review the animal models for DMD, the pros and cons of each model system, and the history and progress of preclinical DMD gene therapy research in the animal models. We also discuss the current and emerging challenges in this field and ways to address these challenges using animal models, in particular cDMD dogs.
Source: Disease Models & Mechanisms
DOI: 10.1242/dmm.018424
Authors: Joe W. McGreevy, Chady H. Hakim, Mark A. McIntosh, Dongsheng Duan
Institution: University of Missouri
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,750
Funders: U.S. Department of Defense, Muscular Dystrophy Association, Parent Project Muscular Dystrophy, University of Missouri, National Institutes of Health, National Heart, Lung, and Blood Institute, National Institute of Neurological Disorders and Stroke, National Institute of Arthritis and Musculoskeletal and Skin Diseases
OpenAlex ID: W2097136884