Cited by 121Virus-based gene therapy researchRNA Interference and Gene DeliveryViral Infectious Diseases and Gene Expression in Insects

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Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene

Halbert, Christine L., Allen, James M., Miller, A. Dusty·2002·Nature Biotechnology

DOI 10.1038/nbt0702-697

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Abstract

The small packaging capacity of adeno-associated virus (AAV) vectors limits the utility of this promising vector system for transfer of large genes. We explored the possibility that larger genes could be reconstituted following homologous recombination between AAV vectors carrying overlapping gene fragments. An alkaline phosphatase (AP) gene was split between two such AAV vectors (rec vectors) and packaged using AAV2 or AAV6 capsid proteins. Rec vectors having either capsid protein recombined to express AP in cultured cells at about 1-2% of the rate observed for an intact vector. Surprisingly, the AAV6 rec vectors transduced lung cells in mice almost as efficiently as did an intact vector, with 10% of airway epithelial cells, the target for treatment of cystic fibrosis (CF), being positive. Thus AAV rec vectors may be useful for diseases such as CF that require transfer of large genes.

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Authors: Halbert, C. L., Allen, J. M., & Miller, A. D.
Affiliations: Fred Hutch Cancer Center (US)
Corresponding author: Miller, A. Dusty
ORCID: Halbert, Christine L.
Allen, James M.
Miller, A. Dusty
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2002-07
Volume: 20
Issue: 7
Pages: 697-701
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt0702-697
OpenAlex: W2042805311
Cited by: 121
References: 25
Citations by year: 2026: 3 · 2025: 3 · 2024: 3 · 2023: 2 · 2022: 6 · 2021: 2 · 2020: 5 · 2019: 3 · 2018: 4 · 2017: 7 · 2016: 3 · 2015: 9 · 2014: 5 · 2013: 5 · 2012: 5
FWCI: 2.65
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
Locations: Nature Biotechnology (publishedVersion)
PubMed (publishedVersion)
Funders: Ministry of Earth Sciences
Ministry of Education, India
National Institutes of Health
National Heart, Lung, and Blood Institute (U01HL066947, HL-66947)
National Institute of Diabetes and Digestive and Kidney Diseases (DK-47754, P30DK047754)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
Keywords: adeno-associated virus vectors, homologous recombination, alkaline phosphatase reporter, airway epithelial cells, cystic fibrosis gene therapy
MeSH terms: Dependovirus, Animals, Gene Expression Regulation, Genetic Vectors, Humans, Lung, Mice, Inbred C57BL, Recombination, Genetic, Transduction, Genetic, Respiratory Mucosa, Capsid Proteins, Mice

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Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene

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