Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter

Gill, DR; Smyth, SE; Goddard, CA; Pringle, IA; Higgins, CF; Colledge, WH; Hyde, SC

doi:10.1038/sj.gt.3301561

Cited by 214RNA Interference and Gene DeliveryVirus-based gene therapy researchAnimal Genetics and Reproduction

Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter

Gill, DR, Smyth, SE, Goddard, CA, Pringle, IA, Higgins, CF, Colledge, WH, Hyde, SC·2001·Gene Therapy

DOI 10.1038/sj.gt.3301561

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Abstract

For effective gene therapy of chronic disease, persistent transgene expression at therapeutic levels is required. Clinical studies of airway gene transfer in patients with cystic fibrosis (CF) have resulted in short-lived transgene expression. We used intra-nasal dosing of naked plasmid DNA to the murine lung as a model for investigating the duration of airway gene transfer from a series of reporter expression plasmids. Transgene expression was transient when mediated by the viral promoters CMV, RSV and SV40, falling to less than 10% of peak expression after 2 weeks, although the presence of the adenoviral E4ORF3 gene in cis, resulted in extended duration of reporter activity from the CMV promoter. Transient expression from these promoters was not due to loss of the vector as determined by quantitative TaqMan PCR analysis. However, use of the promoters from the human polybiquitin C (UbC) and the elongation factor 1alpha (EF1alpha) genes resulted in persistent gene expression in the mouse lung. The UbC promoter directed high-level reporter activity which was maintained for up to 8 weeks and was still detectable 6 months after a single administration. Such persistent airway transgene expression from a nonviral vector without the concomitant expression of a potential antigen has not been reported previously. Thus, despite the persistence of vector DNA in vivo, attenuation of promoter function may lead to silencing of transgene expression and careful selection of promoter sequenc…

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“thus providing the relevant advantage of minimizing the potential risk of on-target chromosomal translocations61.”
From Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing (2019) · cites this paper as [61]

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Authors: Gill, D., Smyth, S., Goddard, C., Pringle, I., Higgins, C., Colledge, W., & Hyde, S.
Affiliations: John Radcliffe Hospital (GB)
University of Cambridge (GB)
Hammersmith Hospital (GB)
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2001-10-01
Volume: 8
Issue: 20
Pages: 1539-1546
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/sj.gt.3301561
OpenAlex: W2122003649
Cited by: 214
References: 34
Citations by year: 2026: 6 · 2025: 4 · 2024: 1 · 2023: 1 · 2022: 2 · 2021: 2 · 2020: 3 · 2019: 4 · 2018: 3 · 2017: 6 · 2016: 4 · 2015: 6 · 2014: 9 · 2013: 7 · 2012: 12
FWCI: 3.61
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Funders: Wellcome Trust
Cystic Fibrosis Trust
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Animal Genetics and Reproduction
Keywords: persistent transgene expression, pulmonary gene therapy, promoter selection, cystic fibrosis, promoter silencing, reporter gene expression
MeSH terms: Administration, Inhalation, Animals, Avian Sarcoma Viruses, Cell Line, Cystic Fibrosis, Cytomegalovirus, Female, Gene Expression Regulation, Genetic Vectors, Humans, Luciferases, Lung, Mice, Inbred BALB C, Plasmids, Promoter Regions, Genetic, Simian virus 40, Time Factors, Genetic Therapy, Statistics, Nonparametric, Transgenes, Peptide Elongation Factor 1, Models, Animal, Ubiquitin C, Mice

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Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter

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