期刊
DISEASE MODELS & MECHANISMS
卷 8, 期 3, 页码 195-213出版社
COMPANY BIOLOGISTS LTD
DOI: 10.1242/dmm.018424
关键词
Duchenne muscular dystrophy; Dystrophin; Animal model; Canine DMD; Gene therapy
资金
- National Institutes of Health [AR-49419, HL-91883]
- Department of Defense [MD-13]
- Muscular Dystrophy Association
- Parent Project Muscular Dystrophy
- Jesse's Journey-The Foundation for Gene and Cell Therapy
- Hope for Javier
- Kansas City Area Life Sciences Institute
- University of Missouri
Duchenne muscular dystrophy (DMD) is a progressive musclewasting 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 similar to 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.
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