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Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

Nakai, Hiroyuki, Storm, Theresa A., Kay, Mark A.·2000·Nature Biotechnology
DOI 10.1038/75390
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Abstract

A major shortcoming to the use of adeno-associated virus (rAAV) vectors is their limited packaging size. To overcome this hurdle, we split an expression cassette and cloned it into two separate vectors. The vectors contained either a nuclear localizing Escherichia coli lacZ transgene (nlslacZ) with a splice acceptor, or the human elongation factor 1alpha ( EF1alpha) gene enhancer/promoter(s) (EF1alphaEP) with a splice donor. We co-injected a promoter-less nlslacZ vector with a vector containing either a single EF1alphaEP or a double copy of the EF1alphaEP in a head-to-head orientation, into the portal vein of mice. Gene expression, measured by both transduction efficiency and quantitation of the recombinant protein, was as much as 60-70% of that obtained from mice that received a single vector containing a complete EFalphaEP/nlslacZ expression cassette. This two-vector approach may allow development of gene therapy strategies that will carry exogenous DNA sequences with large therapeutic cDNAs and/or regulatory elements.

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Crossref · 10.1038/75390

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  • All of the methods based on trans-splicing and homologous recombination require multiple vectors, and the efficiency of some is inherently limited by the efficiencies of intron processing or homologous recombination.

    “All of these methods require multiple vectors, and the efficiency of some of these methods is inherently limited by the efficiencies of intron processing or homologous recombination.”

    From Effect of genome size on AAV vector packaging (2010) · cites this paper as [4]

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Research works printed but not linked: 25

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Metadata

Authors
Nakai, H., Storm, T. A., & Kay, M. A.
Venue
Nature Biotechnology
Publisher
Springer Science and Business Media LLC
Published
2000-05
Volume
18
Issue
5
Pages
527-532
ISSN
  • 1087-0156
  • 1546-1696
DOI
10.1038/75390
Cited by
176
References
2
Language
en
Licence
www.springer.com/tdm (tdm)

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