The DOI matches its Crossref record; 332 of the 332 references it cites were found in a registry or an index.
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Open accessAbstract
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.
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Cites
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Cited by
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Duan, D. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.011 · Cited by 489
Self-citationSkeletal muscle fibrosis: an overview
Mahdy, M. A. A. (2019) · Cell and Tissue Research · doi:10.1007/s00441-018-2955-2 · Cited by 363
GPR41 and GPR43 in Obesity and Inflammation – Protective or Causative?
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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., Allais, M., François, V., Dutilleul, M., Malerba, A., et al. (2017) · Nature Communications · doi:10.1038/ncomms16105 · Cited by 246
Genetically engineered pigs as models for human disease
Perleberg, C., Kind, A., & Schnieke, A. (2018) · Disease Models & Mechanisms · doi:10.1242/dmm.030783 · Cited by 222
Metadata
- Authors
- McGreevy, J. W., Hakim, C. H., McIntosh, M. A., & Duan, D.
- Venue
- Disease Models & Mechanisms
- Publisher
- The Company of Biologists
- Published
- 2015-03-01
- Volume
- 8
- Issue
- 3
- Pages
- 195-213
- ISSN
- 1754-8403 (ISSN-L)
- 1754-8411
- OpenAlex
- W2097136884
- Cited by
- 512
- References
- 332
- FWCI
- 17.88
- Type
- journal article
- Language
- en
- Open access
- gold
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