The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro
Open accessAbstract
We have characterized the ability of adeno-associated virus (AAV) serotypes 1-9 in addition to nineteen novel vectors isolated from various tissues, to transduce mouse and human ciliated airway epithelium (HAE). Vectors expressing alpha-1-antitrypsin (AAT) and beta-galactosidase were co-instilled into the mouse lung. Of all the vectors tested rh.64R1, AAV5 and AAV6 were the most efficient. The high transduction observed in mouse was reproduced in HAE cell cultures for both rh.64R1 and AAV6 but not for AAV5. Since AAV6 was the most efficient vector in mouse and HAE we also tested the transduction efficiencies of the AAV6 singleton vectors (i.e., AAV6 variants with targeted mutations) in these models. Of these, AAV6.2 transduced mouse airway epithelium and HAE with greater efficiency than all other AAV vectors tested. We demonstrated that AAV6.2 exhibits improved transduction efficiency compared to previously reported AAVs in mouse airways and in culture models of human airway epithelium and that this vector requires further development for preclinical and clinical testing.
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Cites
Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA
Riordan, J. R., Rommens, J. M., Kerem, B.-S., Alon, N., Rozmahel, R., Grzelczak, Z., Zielenski, J., Lok, S., Plavsic, N., Chou, J.-L., et al. (1989) · Science · doi:10.1126/science.2475911 · Cited by 7,418
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New recombinant serotypes of AAV vectors
Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487
Self-citationZhang, L., Bukreyev, A., Thompson, C. I., Watson, B., Peeples, M. E., Collins, P. L., & Pickles, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.2.1113-1124.2005 · Cited by 302
Self-citationHeparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
Vandenberghe, L. H., Wang, L., Somanathan, S., Zhi, Y., Figueredo, J., Calcedo, R., Sanmiguel, J., Desai, R. A., Chen, C. S., Johnston, J., et al. (2006) · Nature Medicine · doi:10.1038/nm1445 · Cited by 208
Self-citationWang, L., Calcedo, R., Nichols, T. C., Bellinger, D. A., Dillow, A., Verma, I. M., & Wilson, J. M. (2005) · Blood · doi:10.1182/blood-2004-10-3867 · Cited by 173
Self-citationThe Promise of Gene Therapy For The Treatment of α-1 Antitrypsin Deficiency
Cruz, P. E., Mueller, C., & Flotte, T. R. (2007) · Pharmacogenomics · doi:10.2217/14622416.8.9.1191 · Cited by 10
Cited by
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
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
Weinmann, J., Weis, S., Sippel, J., Tulalamba, W., Remes, A., El Andari, J., Herrmann, A.-K., Pham, Q. H., Borowski, C., Hille, S., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-19230-w · Cited by 259
The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice
Bell, C. L., Vandenberghe, L. H., Bell, P., Limberis, M. P., Gao, G.-P., Van Vliet, K., Agbandje-McKenna, M., & Wilson, J. M. (2011) · Journal of Clinical Investigation · doi:10.1172/jci57367 · Cited by 190
Self-citationControlling AAV Tropism in the Nervous System with Natural and Engineered Capsids
Castle, M. J., Turunen, H. T., Vandenberghe, L. H., & Wolfe, J. H. (2016) · Methods in Molecular Biology · doi:10.1007/978-1-4939-3271-9_10 · Cited by 151
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Metadata
- Authors
- Limberis, M. P., Vandenberghe, L. H., Zhang, L., Pickles, R. J., & Wilson, J. M.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2009-02
- Volume
- 17
- Issue
- 2
- Pages
- 294-301
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2072771134
- Cited by
- 184
- References
- 7
- FWCI
- 5.45
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- The paper
- Limberis, M. P., Vandenberghe, L. H., Zhang, L., Pickles, R. J., & Wilson, J. M. (2009). Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro. Molecular Therapy. https://doi.org/10.1038/mt.2008.261
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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).
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