Helper‐independent and AAV‐ITR‐independent chromosomal integration of double‐stranded linear DNA vectors in mice

Nakai, Hiroyuki; Montini, Eugenio; Fuess, Sally; Storm, Theresa A; Meuse, Leonard; Finegold, Milton; Grompe, Markus; Kay, Mark A

doi:10.1016/s1525-0016(02)00023-0

Cited by 52Virus-based gene therapy researchRNA Interference and Gene DeliveryAnimal Genetics and Reproduction

Helper‐independent and AAV‐ITR‐independent chromosomal integration of double‐stranded linear DNA vectors in mice

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Nakai, Hiroyuki, Montini, Eugenio, Fuess, Sally, Storm, Theresa A, Meuse, Leonard, Finegold, Milton, Grompe, Markus, Kay, Mark A·2003·Molecular Therapy

DOI 10.1016/s1525-0016(02)00023-0

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Abstract

Nonviral plasmid DNA is a promising vector for achieving ex vivo and in vivo gene transfer. However, transgene expression is usually transient, especially in dividing target cells due to loss of vector genomes. Here we describe the use of naked double-stranded (ds) linear DNA as a way to insert exogenous DNA sequences into chromosomes of mouse hepatocytes in vivo, without helper components such as integrase or transposase. We constructed ds linear DNA vectors with or without adeno-associated virus inverted terminal repeats (AAV-ITRs), introduced them into mouse hepatocytes in vivo using a hydrodynamics-based transfection technique, and analyzed for vector genome integration in various ways. Surprisingly, these linear DNA molecules integrated in mouse hepatocytes in vivo at a level of 0.3-0.5 vector genome, or more, per diploid genomic equivalent irrespective of the AAV-ITR sequences. Our results establish a novel and simple way to engineer chromosomes in vivo and provide further insights into the mechanisms of recombinant AAV vector integration in vivo. In addition, they may provide a clue for developing new nonviral integrating gene delivery vector systems.

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doi:10.1016/S1525-0016(02)00023-0 ARTICLE Helper-Independent and AAV-ITR-Independent Chromosomal Integration of Double-Stranded Linear DNA Vectors in Mice
Nakai, H., Montini, E., Fuess, S., Storm, T. A., Meuse, L. A., Finegold, M. J., Grompe, M., & Kay, M. A. (2014)
991. Helper-Independent Chromosomal Integration of Naked Double-Stranded Linear DNA Vectors in Mouse Liver In Vivo
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Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection
Wang, Z., Troilo, P. J., Griffiths, T. G., Harper, L. B., Barnum, A. B., Pacchione, S. J., Pauley, C. J., Lebron, J. A., Wolf, J. J., & Ledwith, B. J. (2021) · Gene Therapy · doi:10.1038/s41434-021-00278-2 · Cited by 15 · see citing works
Requirements for Adeno-Associated Virus-Derived Non-Viral Vectors to Achieve Stable and Site-Specific Integration of Plasmid DNA in Liver Carcinoma Cells
Steigerwald, R., Rabe, C., Schmitz, V., Schmidt‐Wolf, I. G. H., Alt, M., & Caselmann, W. H. (2003) · Digestion · doi:10.1159/000073221 · Cited by 6 · see citing works

Authors: Nakai, H., Montini, E., Fuess, S., Storm, T. A., Meuse, L., Finegold, M., Grompe, M., & Kay, M. A.
Affiliations: Stanford Medicine (US)
Texas Children's Hospital (US)
Oregon Health & Science University (US)
ORCID: Nakai, Hiroyuki
Montini, Eugenio
Storm, Theresa A
Finegold, Milton
Grompe, Markus
Kay, Mark A
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2003-01
Volume: 7
Issue: 1
Pages: 101-111
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1016/s1525-0016(02)00023-0
OpenAlex: W2083399907
Cited by: 52
References: 33
Citations by year: 2023: 1 · 2022: 1 · 2021: 3 · 2020: 1 · 2018: 1 · 2017: 1 · 2016: 1 · 2015: 2 · 2014: 1 · 2013: 1 · 2012: 3
FWCI: 3.50
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 5028 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
Funders: National Heart, Lung, and Blood Institute (HL64274, R01HL064274)
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 › Genetics › Animal Genetics and Reproduction
Keywords: chromosomal integration, non-viral gene delivery, hepatocytes, hydrodynamic gene transfer, transgene expression, in vivo gene delivery
MeSH terms: Dependovirus, Animals, Base Sequence, Chromosomes, DNA, Mice, Inbred C57BL, Molecular Sequence Data, Blotting, Southern, Hepatocytes, Mice

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Helper‐independent and AAV‐ITR‐independent chromosomal integration of double‐stranded linear DNA vectors in mice

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