Risk assessment in skin gene therapy: viral–cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors
Open accessAbstract
Cutaneous gene therapy can be envisioned through the use of keratinocyte stem cell clones in which retroviral genotoxic risks can be pre-assessed. While transactivation of cellular genes by the retroviral long terminal repeat enhancer has been proven in experimental and clinical settings, the formation of chimeric viral-cellular transcripts originated by the inefficient termination (read-through) of retroviral transcripts remains to be studied in depth. We now demonstrate the widespread presence of viral-cellular fusion transcripts derived from integrated proviruses in keratinocytes transduced with self-inactivating (SIN) retroviral vectors. We have detected high molecular weight RNAs in northern blot analysis of retroviral vector expression in individual cell clones. Characterization of some of these transcripts revealed that they originate from genes located at the proviral integration sites. One class of transcripts corresponds to fusions of the viral vectors with intronic sequences, terminating at cryptic polyadenylation sites located in introns. A second class comprises fusion transcripts with coding sequences of genes at the integration sites. These are generated through splicing from a cryptic, not previously described donor site in the lentiviral vectors to exons of cellular genes, and have the potential to encode unintended open reading frames, although they are downregulated by cellular mechanisms. Our data contribute to a better understanding of the impact of SIN lentiviral vector integration on cellular gene transcription, and will be helpful in improving the design of this type of vectors.
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Some viral vectors are considered risky for potential oncogenesis.
“Although many viral vectors are safe and effective delivery vehicles for clinical gene therapy, some viral vectors are considered risky for potential oncogenesis.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [29]
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- Authors
- Almarza, D., Bussadori, G., Navarro, M., Mavilio, F., Larcher, F., & Murillas, R.
- Affiliations
- Centre for Biomedical Network Research on Rare Diseases (ES)
- University of Modena and Reggio Emilia (IT)
- Corresponding author
- Larcher, F
- Murillas, R
- Venue
- Gene Therapy
- Published
- 2011-03-03
- Volume
- 18
- Issue
- 7
- Pages
- 674-681
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2070082521
- Cited by
- 56
- References
- 24
- Citations by year
- 2026: 2 · 2025: 5 · 2024: 3 · 2023: 8 · 2022: 1 · 2021: 1 · 2020: 3 · 2019: 2 · 2018: 1 · 2017: 8 · 2015: 2 · 2014: 3 · 2013: 6 · 2012: 7
- FWCI
- 4.00
- Type
- journal article
- Language
- en
- Open access
- green
- hdl.handle.net
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- IRIS UNIMORE (University of Modena and Reggio Emilia) (submittedVersion, cc-by)
- Funders
- Comunidad de Madrid
- Ministerio de Ciencia e Innovación
- Instituto de Salud Carlos III
- 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 › CRISPR and Genetic Engineering
- Keywords
- skin gene therapy, keratinocytes, self-inactivating lentiviral vectors, retroviral integration sites, splicing, vector design
- MeSH terms
- Genetic Vectors, Humans, Proviruses, Transcription, Genetic, Transduction, Genetic, Genetic Therapy, Keratinocytes, Lentivirus, Virus Integration, Risk Assessment, Virus Inactivation, Green Fluorescent Proteins, Gene Fusion, Keratin-14
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