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Virus treatment questioned after gene therapy death
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“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 [69]
In the citing paper
“Zuris et al. injected Cas9:sgRNA complexes targeting GFP gene into the cochlea of transgenic Atoh1-GFP mice. This system resulted in ~20% Cas9-mediated genome modification in hair cells [69].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [69]
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Metadata
- Authors
- Lehrman, S.
- Corresponding author
- Lehrman, Sally
- ORCID
- Lehrman, Sally
- Venue
- Nature
- Published
- 1999-10-07
- Volume
- 401
- Issue
- 6753
- Pages
- 517-518
- ISSN
- 0028-0836 (ISSN-L)
- 1476-4687
- OpenAlex
- W1606853210
- Cited by
- 576
- References
- 0
- Citations by year
- 2026: 6 · 2025: 9 · 2024: 11 · 2023: 15 · 2022: 17 · 2021: 12 · 2020: 18 · 2019: 16 · 2018: 18 · 2017: 14 · 2016: 14 · 2015: 24 · 2014: 30 · 2013: 23 · 2012: 31
- Type
- other
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature (publishedVersion)
- PubMed (publishedVersion)
- RePEc: Research Papers in Economics (submittedVersion)
- DigitalGeorgetown (Georgetown University Library) (submittedVersion)
- Funders
- Brigham and Women's Hospital
- University of East Anglia
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Nursing › Issues, ethics and legal aspects › Biomedical Ethics and Regulation
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Keywords
- gene therapy, viral vectors, recombinant adeno-associated virus, clinical trial safety
- MeSH terms
- Adenoviridae, Adult, Fever, Genetic Vectors, Humans, Male, Ornithine Carbamoyltransferase, Thrombosis, Genetic Therapy, Clinical Trials, Phase I as Topic, Fatal Outcome, Risk Assessment, Ornithine Carbamoyltransferase Deficiency Disease, Advisory Committees, Government Regulation, Federal Government, Research Subjects
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