The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.
Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs
Open accessAbstract
Duchenne (DMD) and golden retriever (GRMD) muscular dystrophy are caused by genetic mutations in the dystrophin gene and afflict striated muscles. We investigated systemic gene delivery in 4-day-old GRMD dogs given a single intravenous injection of an AAV9 vector (1.5 x 10(14) vector genomes/kg) carrying a human codon-optimized human mini-dystrophin gene under control of the cytomegalovirus (CMV) promoter. One of the three treated dogs was euthanized 9 days later due to pre-existing conditions. Scattered mini-dystrophin-positive myofibers were seen by immunofluorescent (IF) staining in numerous muscles. At the end of the 16-week study, the other two dogs showed generalized muscle expression of mini-dystrophin in ~15% to nearly 100% of myofibers. Western blot and vector DNA quantitative PCR results agreed with the IF data. Delayed growth and pelvic limb muscle atrophy and contractures were seen several weeks after vector delivery. T-2 weighted magnetic resonance imaging (MRI) at 8 weeks showed increased signal intensity compatible with inflammation in several pelvic limb muscles. This marked early inflammatory response raised concerns regarding methodology. Use of the ubiquitous CMV promoter, extra-high vector dose, and marked expression of a human protein in canine muscles may have contributed to the pathologic changes seen in the pelvic limbs.
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Dystrophin: the protein product of the Duchenne muscular dystrophy locus
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X chromosome-linked muscular dystrophy (mdx) in the mouse
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Self-citationThe homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs
Cooper, B. J., Winand, N. J., Stedman, H., Valentine, B. A., Hoffman, E. P., Kunkel, L. M., Scott, M.-O., Fischbeck, K. H., Kornegay, J. N., Avery, R. J., et al. (1988) · Nature · doi:10.1038/334154a0 · Cited by 428
Self-citationHeparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
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Lee, K., Conboy, M., Park, H. M., Jiang, F., Kim, H. J., Dewitt, M. A., Mackley, V. A., Chang, K., Rao, A., Skinner, C., et al. (2017) · Nature Biomedical Engineering · doi:10.1038/s41551-017-0137-2 · Cited by 755
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
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
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
Bevan, A. K., Duque, S., Foust, K. D., Morales, P. R., Braun, L., Schmelzer, L., Chan, C. M., McCrate, M., Chicoine, L. G., Coley, B. D., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.157 · Cited by 337
Metadata
- Authors
- Kornegay, J. N., Li, J., Bogan, J. R., Bogan, D. J., Chen, C., Zheng, H., Wang, B., Qiao, C., Howard, J. F., & Xiao, X.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2010-08
- Volume
- 18
- Issue
- 8
- Pages
- 1501-1508
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1968543820
- Cited by
- 158
- References
- 6
- FWCI
- 8.05
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Kornegay, J. N., Li, J., Bogan, J. R., Bogan, D. J., Chen, C., Zheng, H., Wang, B., Qiao, C., Howard, J. F., & Xiao, X. (2010). Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs. Molecular Therapy. https://doi.org/10.1038/mt.2010.94
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