High-dose AAV gene therapy deaths
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Viral delivery platforms can be limited by pre-existing viral immunity, transgene size constraints, undesired vector integration, and adverse events related to the viral vector.
“Although viral vectors are a promising approach to deliver gene editing technology to the CNS, viral delivery platforms can be limited by pre-existing viral immunity, transgene size constraints, undesired vector integration, and adverse events related to the viral vector (10–12).”
From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [12]
High dose systemic rAAV poses concerns over dosage related hepatotoxicity.
“High dose intravenous AAV9 (2×1014 vector genomes (vg)/kg) in NHP resulted in marked transaminitis and acute liver failure (66), posing concerns over dosage related hepatotoxicity (67).”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [67]
Cited by
Sorted by citations
Adeno-Associated Virus Toolkit to Target Diverse Brain Cells
Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148 · see citing works
Journey to the Center of the Cell: Tracing the Path of AAV Transduction
Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2020.09.010 · Cited by 122 · see citing works
Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease
Palanki, R., Bose, S. K., Dave, A., White, B. M., Berkowitz, C., Luks, V., Yaqoob, F., Han, E., Swingle, K. L., Menon, P., et al. (2023) · ACS Nano · doi:10.1021/acsnano.3c02268 · Cited by 77 · see citing works
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
Wagner, D. L., Peter, L., & Schmueck-Henneresse, M. (2021) · Gene Therapy · doi:10.1038/s41434-021-00232-2 · Cited by 63 · see citing works
Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies
Chu, W. S., & Ng, J. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.658038 · Cited by 59 · see citing works
Rode, L., Bär, C., Groß, S., Rossi, A., Meumann, N., Viereck, J., Abbas, N., Xiao, K., Riedel, I., Gietz, A., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.07.003 · Cited by 48 · see citing works
Costa-Verdera, H., Meneghini, V., Fitzpatrick, Z., Abou Alezz, M., Fabyanic, E., Huang, X., Dzhashiashvili, Y., Ahiya, A., Mangiameli, E., Valeri, E., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-58778-3 · Cited by 32 · see citing works
Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps
Brimble, M. A., Cheng, P.-H., Winston, S. M., Reeves, I. L., Souquette, A., Spence, Y., Zhou, J., Wang, Y.-D., Morton, C. L., Valentine, M., et al. (2022) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2022.01.008 · Cited by 30 · see citing works
Zhang, X., Chai, Z., Lee Dobbins, A., Itano, M. S., Askew, C., Miao, Z., Niu, H., Samulski, R. J., & Li, C. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2021.121340 · Cited by 23 · see citing works
Glenn, J. D., Negash, H., Henry, W., Qian, R., Liu, Y., Danos, O., Bruder, J. T., & Karumuthil-Melethil, S. (2024) · Cellular Immunology · doi:10.1016/j.cellimm.2024.104823 · Cited by 6 · see citing works
AAV vectors trigger DNA damage responses and STING-dependent inflammation in human CNS cells
Costa-Verdera, H., Meneghini, V., Fitzpatrick, Z., Alezz, M. A., Fabyanic, E., Huang, X., Dzhashiashvili, Y., Ahiya, A., Mangiameli, E., Valeri, E., et al. (2024) · doi:10.21203/rs.3.rs-4171795/v1 · Cited by 32 · see citing works
- Venue
- Nature Biotechnology
- Published
- 2020-08
- Volume
- 38
- Issue
- 8
- Pages
- 910-910
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W4205532486
- Cited by
- 234
- References
- 0
- Citations by year
- 2026: 26 · 2025: 36 · 2024: 37 · 2023: 47 · 2022: 44 · 2021: 36 · 2020: 8
- Type
- other
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- AAV gene therapy, X-linked myotubular myopathy, liver dysfunction, sepsis, gene therapy safety, rare neuromuscular diseases
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
