The DOI matches its Crossref record; 5 of the 5 references it cites were found in a registry or an index.
A largely random AAV integration profile after LPLD gene therapy
Abstract
The clinical application of adeno-associated virus vectors (AAVs) is limited because of concerns about AAV integration-mediated tumorigenicity. We performed integration-site analysis after AAV1-LPL(S447X) intramuscular injection in five lipoprotein lipase-deficient subjects, revealing random nuclear integration and hotspots in mitochondria. We conclude that AAV integration is potentially safe and that vector breakage and integration may occur from each position of the vector genome. Future viral integration-site analyses should include the mitochondrial genome.
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“After transduction, AAV DNA remains mostly episomal besides integrating into hotspots in mitochondrial DNA and a specific location on chromosome 19 termed AAVS1 [14,15,16].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [14]
Citations
Cites
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High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
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Cited by
Sorted by citations
Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444
Viral Delivery Systems for CRISPR
Xu, C. L., Ruan, M. Z. C., Mahajan, V. B., & Tsang, S. H. (2019) · Viruses · doi:10.3390/v11010028 · Cited by 277
Genetics and causality of triglyceride-rich lipoproteins in atherosclerotic cardiovascular disease
Rosenson, R. S., Davidson, M. H., Hirsh, B. J., Kathiresan, S., & Gaudet, D. (2014) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2014.09.042 · Cited by 260
Self-citationBasic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balakrishnan, B., & Jayandharan, G. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140302193709 · Cited by 225
Gil-Farina, I., Fronza, R., Kaeppel, C., Lopez-Franco, E., Ferreira, V., D'Avola, D., Benito, A., Prieto, J., Petry, H., Gonzalez-Aseguinolaza, G., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.52 · Cited by 179
Self-citation
Metadata
- Authors
- Kaeppel, C., Beattie, S. G., Fronza, R., van Logtenstein, R., Salmon, F., Schmidt, S., Wolf, S., Nowrouzi, A., Glimm, H., von Kalle, C., Petry, H., Gaudet, D., & Schmidt, M.
- Venue
- Nature Medicine
- Published
- 2013-06-16
- Volume
- 19
- Issue
- 7
- Pages
- 889-891
- ISSN
- 1078-8956
- 1546-170X
- Cited by
- 176
- References
- 5
- Language
- en
- Licence
- www.springer.com/tdm (tdm)
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