The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.
Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6
Open accessAbstract
Vectors based on recombinant adeno-associated viruses (rAAV) have emerged as tools of choice for gene transfer to skeletal muscle. rAAV vectors demonstrate efficient, safe, and stable transduction. Multiple serotypes of AAV exist, but vectors based on serotype 2 (rAAV2) are the most thoroughly characterized and frequently employed. Here, we characterize transduction of the skeletal musculature using rAAV vectors pseudotyped with serotype 6 capsid proteins (rAAV6). We demonstrate that rAAV6 vectors can efficiently transduce the skeletal musculature of mice at levels >500-fold higher than is achievable with rAAV2 vectors and can readily saturate individual muscles following direct injection. Further, rAAV6 vectors are capable of transducing the diaphragm and intercostal muscles of mice after a simple injection into the intrathoracic cavity and are capable of widespread transduction throughout the musculature of mice injected in the intraperitoneal space as newborn pups. These results demonstrate that rAAV6 vectors hold great potential for use in gene delivery protocols targeting the skeletal musculature.
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Cites
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Self-citationGene therapy vectors based on adeno-associated virus type 1
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Cited by
Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Naso, M. F., Tomkowicz, B., Perry, W. L., & Strohl, W. R. (2017) · BioDrugs · doi:10.1007/s40259-017-0234-5 · Cited by 1,362
Inagaki, 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
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Metadata
- Authors
- Blankinship, M. J., Gregorevic, P., Allen, J. M., Harper, S. Q., Harper, H., Halbert, C. L., Miller, D. A., & Chamberlain, J. S.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2004-10
- Volume
- 10
- Issue
- 4
- Pages
- 671-678
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1985238823
- Cited by
- 251
- References
- 6
- FWCI
- 9.09
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Blankinship, M. J., Gregorevic, P., Allen, J. M., Harper, S. Q., Harper, H., Halbert, C. L., Miller, D. A., & Chamberlain, J. S. (2004). Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2004.07.016
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