Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction
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Significance: Viral vectors based on the adeno-associated virus (AAV) are at the forefront of human gene therapy; yet, the molecular biology of these vectors is largely unexplored. The aim of this work was to unravel, in a systematic manner, the effect of the individual knockdown of over 18,000 human genes on the efficiency of AAV-mediated transduction. The results obtained connect AAV biology to the processing of cellular DNA damage and pave the way to practical applications to improve therapeutic gene transfer.
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The knockdown of chromatin assembly factor 1 increased rAAV transduction in HeLa cells.
“Besides, the knockdown of chromatin assembly factor 1 increased rAAV transduction in HeLa cells [242].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [242]
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- [52]Ayuso E, et al. (2010) High AAV vector purity results in serotypeand tissueindependent enhancement of transduction efficiency. Gene Ther 17(4):503 -510.
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Buck, T., & Wijnholds, J. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21124197 · Cited by 92 · see citing works
“Besides, the knockdown of chromatin assembly factor 1 increased rAAV transduction in HeLa cells [242].”
BackgroundIntramuscular Delivery of Gene Therapy for Targeting the Nervous System
Tosolini, A. P., & Sleigh, J. N. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00129 · Cited by 32 · see citing works
- Authors
- Mano, M., Ippodrino, R., Zentilin, L., Zacchigna, S., & Giacca, M.
- Affiliations
- International Centre for Genetic Engineering and Biotechnology (IT)
- University of Trieste (IT)
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2015-08-24
- Volume
- 112
- Issue
- 36
- Pages
- 11276-11281
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC4568673
- OpenAlex
- W2108988659
- Cited by
- 39
- References
- 54
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 8 · 2023: 5 · 2022: 3 · 2021: 3 · 2020: 5 · 2019: 7 · 2018: 4 · 2017: 1 · 2016: 1
- FWCI
- 1.37
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- ArTS Archivio della ricerca di Trieste (University of Trieste https://www.units.it/) (submittedVersion, other-oa)
- Research Portal (King's College London) (submittedVersion)
- Funders
- EC | European Research Council (250124)
- Fondazione Telethon (GGP11068)
- 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 › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- adeno-associated virus, AAV transduction, host restriction factors, siRNA screen, gene delivery, in vivo gene delivery, cell cycle checkpoints, liver gene delivery, viral vectors
- MeSH terms
- Dependovirus, Animals, Cell Line, Genetic Vectors, HeLa Cells, Humans, Liver, Luminescent Proteins, Microscopy, Fluorescence, Transduction, Genetic, Genetic Therapy, Gene Expression, Genome, Human, Microscopy, Confocal, Transgenes, Reverse Transcriptase Polymerase Chain Reaction, RNA Interference, Cell Line, Tumor, Green Fluorescent Proteins, Mice, Host-Pathogen Interactions, HEK293 Cells
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