The DOI matches its Crossref record; 11 of the 11 references it cites were found in a registry or an index.
From Virus Evolution to Vector Revolution: Use of Naturally Occurring Serotypes of Adeno-associated Virus (AAV) as Novel Vectors for Human Gene Therapy
Abstract
Gene transfer vectors based on the human adeno-associated virus serotype 2 (AAV-2) have been developed and tested in pre-clinical studies for almost 20 years, and are currently being evaluated in clinical trials. So far, all these studies have provided evidence that AAV-2 vectors possess many properties making them very attractive for therapeutic gene delivery to humans, such as a lack of pathogenicity or toxicity, and the ability to confer long-term gene expression. However, there is concern that two restrictions of AAV-2 vectors might limit their clinical use in humans. First, these vectors are rather inefficient at transducing some cells of therapeutic interest, such as liver and muscle cells. Second, gene transfer might be hampered by neutralizing anti-AAV-2 antibodies, which are highly prevalent in the human population. In efforts to overcome both limitations, an increasing number of researchers are now focusing on the seven other naturally occurring serotypes of AAV (AAV-1 and AAV-3 to -8), which are structurally and functionally different from AAV-2. To this end, several strategies have been devised to cross-package an AAV-2 vector genome into the capsids of the other AAV serotypes, resulting in a new generation of "pseudotyped" AAV vectors. In vitro and in vivo, these novel vectors were shown to have a host range different from AAV-2, and to escape the anti-AAV-2 immune response, thus underscoring the great potential of this approach. Here the biology of the eight AAV serotypes is summarized, existing technology for pseudotyped AAV vector production is described, initial results from pre-clinical evaluation of the vectors are reviewed, and finally, the prospects of these promising novel tools for human gene therapy are discussed.
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Citations
Cites
Gene therapy restores vision in a canine model of childhood blindness
Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202 · cited 3×
“A typical example is Leber's congenital amaurosis (LCA), the earliest and most severe form of retinal degeneration.”
BackgroundInfluentialGene therapy vectors based on adeno-associated virus type 1
Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., & Wilson, J. M. (1999) · Journal of Virology · doi:10.1128/jvi.73.5.3994-4003.1999 · Cited by 541
Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
Bartlett, J. S., Wilcher, R., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.6.2777-2785.2000 · Cited by 532 · cited 1×
“This is in contrast to AAV-2 [Bartlett et al., 2000] and thus again highlights the diversity amongst AAV serotypes.”
ResultDuan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K. J., & Engelhardt, J. F. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8568-8577.1998 · Cited by 522
Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus
Duan, D., Yue, Y., Yan, Z., Yang, J., & Engelhardt, J. F. (2000) · Journal of Clinical Investigation · doi:10.1172/jci8317 · Cited by 391
Chao, H., Liu, Y., Rabinowitz, J., Li, C., Samulski, R. J., & Walsh, C. E. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0219 · Cited by 366
Grimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00095-9 · Cited by 344
Self-citationYan, Z., Zak, R., Luxton, G. W. G., Ritchie, T. C., Bantel-Schaal, U., & Engelhardt, J. F. (2002) · Journal of Virology · doi:10.1128/jvi.76.5.2043-2053.2002 · Cited by 222
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Grimm, D., Kern, A., Pawlita, M., Ferrari, F. K., Samulski, R. J., & Kleinschmidt, J. A. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300946 · Cited by 195 · cited 1×
“This would underscore the general idea that AAV vector titers are mainly determined by expression of capsid proteins from the helper [Grimm et al., 1998,1999].”
BackgroundSelf-citation
Cited by
Design and development of polymers for gene delivery
Pack, D. W., Hoffman, A. S., Pun, S., & Stayton, P. S. (2005) · Nature Reviews Drug Discovery · doi:10.1038/nrd1775 · Cited by 2,568
Progress and problems with the use of viral vectors for gene therapy
Thomas, C. E., Ehrhardt, A., & Kay, M. A. (2003) · Nature Reviews Genetics · doi:10.1038/nrg1066 · Cited by 2,501
Self-citationFatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
Grimm, D., Streetz, K. L., Jopling, C. L., Storm, T. A., Pandey, K., Davis, C. R., Marion, P., Salazar, F., & Kay, M. A. (2006) · Nature · doi:10.1038/nature04791 · Cited by 1,677
Self-citationGrimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696
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
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Metadata
- Authors
- Grimm, D., & Kay, M.
- Venue
- Current Gene Therapy
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2003-08-01
- Volume
- 3
- Issue
- 4
- Pages
- 281-304
- ISSN
- 1566-5232 (ISSN-L)
- OpenAlex
- W2088555526
- Cited by
- 350
- References
- 11
- FWCI
- 16.58
- Type
- journal article
- Language
- en
- Open access
- closed
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