The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Sustained AAV-mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression
Open accessAbstract
Adeno-associated virus-based vector (AAV)-mediated gene delivery has been successful in some animal models of human disease such as the mdx mouse model of human Duchenne muscular dystrophy (DMD). However, recent evidence of immune-mediated loss of vector persistence in dogs and humans suggests that immune modulation might be necessary to achieve successful long-term transgene expression in these species. We have previously demonstrated that direct intramuscular injection of AAV2 or AAV6 in wild-type random-bred dogs resulted in a robust immune response to capsid or capsid-associated proteins. We now demonstrate that a brief course of immunosuppression with a combination of anti-thymocyte globulin (ATG), cyclosporine (CSP), and mycophenolate mofetil (MMF) is sufficient to permit long-term and robust expression of a canine micro-dystrophin (c-micro-dys) transgene in the skeletal muscle of a dog model for DMD (canine X-linked muscular dystrophy, or cxmd dog) and that its expression restored localization of components of the dystrophin-associated protein complex at the muscle membrane. This protocol has potential applications to human clinical trials to enhance AAV-mediated therapies.
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Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
England, S. B., Nicholson, L. V. B., Johnson, M. A., Forrest, S. M., Love, D. R., Zubrzycka-Gaarn, E. E., Bulman, D. E., Harris, J. B., & Davies, K. E. (1990) · Nature · doi:10.1038/343180a0 · Cited by 623
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
Gregorevic, P., Blankinship, M. J., Allen, J. M., Crawford, R. W., Meuse, L., Miller, D. G., Russell, D. W., & Chamberlain, J. S. (2004) · Nature Medicine · doi:10.1038/nm1085 · Cited by 620
Self-citationInagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.014 · Cited by 610
Cited by
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955
State-of-the-art gene-based therapies: the road ahead
Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 666
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
McGreevy, J. W., Hakim, C. H., McIntosh, M. A., & Duan, D. (2015) · Disease Models & Mechanisms · doi:10.1242/dmm.018424 · Cited by 512
The AAV Vector Toolkit: Poised at the Clinical Crossroads
Asokan, A., Schaffer, D. V., & Jude Samulski, R. (2012) · Molecular Therapy · doi:10.1038/mt.2011.287 · Cited by 465
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
Metadata
- Authors
- Wang, Z., Kuhr, C. S., Allen, J. M., Blankinship, M., Gregorevic, P., Chamberlain, J. S., Tapscott, S. J., & Storb, R.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2007-06
- Volume
- 15
- Issue
- 6
- Pages
- 1160-1166
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2021202438
- Cited by
- 218
- References
- 4
- FWCI
- 12.06
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Wang, Z., Kuhr, C. S., Allen, J. M., Blankinship, M., Gregorevic, P., Chamberlain, J. S., Tapscott, S. J., & Storb, R. (2007). Sustained AAV-mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression. Molecular Therapy. https://doi.org/10.1038/sj.mt.6300161
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
