Virus-mediated gene delivery for human gene therapy
Abstract
After over 20 years from the first application of gene transfer in humans, gene therapy is now a mature discipline, which has progressively overcome several of the hurdles that prevented clinical success in the early stages of application. So far, the vast majority of gene therapy clinical trials have exploited viral vectors as very efficient nucleic acid delivery vehicles both in vivo and ex vivo. Here we summarize the current status of viral gene transfer for clinical applications, with special emphasis on the molecular properties of the major classes of viral vectors and the information so far obtained from gene therapy clinical trials.
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Adenoviral and adeno-associated-viral (AAV) delivery systems are either associated with immune responses or necessitate multiple treatments due to their transient cellular persistence.
“Among these are the adenoviral and adeno-associated-viral (AAV) delivery systems which, however, are either associated with immune responses or necessitate multiple treatments due to their transient cellular persistence54,55.”
From Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing (2019) · cites this paper as [55]
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Self-citation
- Authors
- Giacca, M., & Zacchigna, S.
- Affiliations
- International Centre for Genetic Engineering and Biotechnology (IT)
- Corresponding author
- Giacca, Mauro
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2012-07
- Volume
- 161
- Issue
- 2
- Pages
- 377-388
- ISSN
- 0168-3659 (ISSN-L)
- OpenAlex
- W2054174437
- Cited by
- 302
- References
- 10
- Citations by year
- 2026: 4 · 2025: 6 · 2024: 20 · 2023: 11 · 2022: 22 · 2021: 24 · 2020: 23 · 2019: 25 · 2018: 22 · 2017: 25 · 2016: 30 · 2015: 36 · 2014: 29 · 2013: 23 · 2012: 2
- FWCI
- 3.55
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Controlled Release (publishedVersion)
- PubMed (publishedVersion)
- ArTS Archivio della ricerca di Trieste (University of Trieste https://www.units.it/) (submittedVersion)
- Funders
- Fondazione Telethon
- 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
- gene therapy, viral vectors, viral gene delivery, nucleic acid delivery, clinical trials, in vivo gene delivery, ex vivo gene transfer
- MeSH terms
- Animals, Genetic Vectors, Humans, Viruses, Genetic Therapy, Gene Transfer Techniques
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