Adverse effects of gene therapy: gene therapy can cause leukaemia: no shock, mild horror but a probe
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Viral vectors have fundamental shortcomings, including risk of carcinogenesis, limited insertion size, immune responses, and difficulty in large-scale production, which severely limit their further applications.
“Although viral vectors have been widely used in the delivery of the CRISPR/Cas9 system in vitro and in vivo, their fundamental shortcomings, such as the risk of carcinogenesis, limited insertion size, immune responses and difficulty in large-scale production, severely limit their further applications.”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [70]
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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,703 · see citing works
Non‐endocrine late complications of bone marrow transplantation in childhood: part II
Leiper, A. D. (2002) · British Journal of Haematology · doi:10.1046/j.1365-2141.2002.03471.x · Cited by 102 · see citing works
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Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities
Li, L., Hu, S., & Chen, X. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2018.04.031 · Cited by 415 · see citing works
Challenges in CRISPR/CAS9 delivery: potential roles of nonviral vectors
Li, L., He, Z.-Y., Wei, X.-W., Gao, G.-P., & Wei, Y.-Q. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.069 · Cited by 202 · see citing works
Reversibly stable thiopolyplexes for intracellular delivery of genes
Cavallaro, G., Campisi, M., Licciardi, M., Ogris, M., & Giammona, G. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.07.027 · Cited by 54 · see citing works
- Authors
- Gore, M. E.
- Affiliations
- Royal Marsden Hospital (GB)
- Corresponding author
- Gore, M E
- Venue
- Gene Therapy
- Published
- 2003-01-01
- Volume
- 10
- Issue
- 1
- Pages
- 4-4
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2069530772
- Cited by
- 53
- References
- 2
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 2 · 2023: 1 · 2022: 1 · 2021: 1 · 2020: 2 · 2019: 4 · 2018: 2 · 2017: 3 · 2016: 1 · 2015: 4 · 2014: 3 · 2012: 2
- Type
- other
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Gene Therapy (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- adverse effects, leukemia, cancer risk, gene therapy clinical trials, pediatric patients, trial suspension, clinical trial safety
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