Tsaregorodtseva, Tatiana S.; Radyukhina, Maria A.; Ayupova, Aisylu I.; Solovyeva, Valeriya V.; Sufianov, Albert A.; Sufianova, Galina Z.; Rizvanov, Albert A. (2026) Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization. International Journal of Molecular Sciences, 27 (11). doi:10.3390/ijms27114818
| Reference Type | Journal (article/letter/editorial) | ||
|---|---|---|---|
| Title | Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization | ||
| Journal | International Journal of Molecular Sciences | ||
| Authors | Tsaregorodtseva, Tatiana S. | Author | |
| Radyukhina, Maria A. | Author | ||
| Ayupova, Aisylu I. | Author | ||
| Solovyeva, Valeriya V. | Author | ||
| Sufianov, Albert A. | Author | ||
| Sufianova, Galina Z. | Author | ||
| Rizvanov, Albert A. | Author | ||
| Year | 2026 (May 27) | Volume | 27 |
| Issue | 11 | ||
| Publisher | MDPI AG | ||
| DOI | doi:10.3390/ijms27114818Search in ResearchGate | ||
| Generate Citation Formats | |||
| Mindat Ref. ID | 20032749 | Long-form Identifier | mindat:1:5:20032749:9 |
| GUID | 0 | ||
| Full Reference | Tsaregorodtseva, Tatiana S.; Radyukhina, Maria A.; Ayupova, Aisylu I.; Solovyeva, Valeriya V.; Sufianov, Albert A.; Sufianova, Galina Z.; Rizvanov, Albert A. (2026) Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization. International Journal of Molecular Sciences, 27 (11). doi:10.3390/ijms27114818 | ||
| Plain Text | Tsaregorodtseva, Tatiana S.; Radyukhina, Maria A.; Ayupova, Aisylu I.; Solovyeva, Valeriya V.; Sufianov, Albert A.; Sufianova, Galina Z.; Rizvanov, Albert A. (2026) Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization. International Journal of Molecular Sciences, 27 (11). doi:10.3390/ijms27114818 | ||
| In | (2026, May) International Journal of Molecular Sciences Vol. 27 (11). MDPI AG | ||
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| Not Yet Imported: Adeno-Associated Virus - book-chapter : 10.1007/978-1-61779-370-7_6 If you would like this item imported into the Digital Library, please contact us quoting Book ID 9781617793691 | |
| Not Yet Imported: Molecular Therapy - journal-article : 10.1038/mt.2014.235 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1128/JVI.05375-11 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1089/hum.2021.124 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1111/cpr.13339 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1089/hum.2022.170 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Horton (2022) J. Neurol. Neurosurg. Psychiatry A Systematic Review of Adeno-Associated Virus Gene Therapies in Neurology: The Need for Consistent Safety Monitoring of a Promising Treatment 93, 1276 | |
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| Not Yet Imported: - journal-article : 10.1007/s00281-022-00964-2 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: Molecular Therapy - Methods & Clinical Development - journal-article : 10.1016/j.omtm.2023.02.015 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.omtm.2024.101244 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.ymthe.2003.08.015 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/S0140-6736(16)30371-3 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.47102/annals-acadmedsg.V37N7p591 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Isildak (2018) Case Rep. Ophthalmol. Med. Therapeutic Effect of Anti-VEGF for Age-Related Macular Degeneration in the Untreated Fellow Eye 2018, 8561895 | |
| Moffit (2025) Mol. Ther. Methods Clin. Dev. Nonclinical Strategies and Considerations to Enable the Redosing of Gene Therapies 33, 101520 | |
| Not Yet Imported: - journal-article : 10.1002/jmr.2824 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: The Journal of Gene Medicine - journal-article : 10.1002/jgm.1161 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1089/hum.2014.082 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1126/scitranslmed.3005795 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1089/hum.2009.157 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: Gene Therapy - journal-article : 10.1038/gt.2017.75 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
![]() | Bertin, Berangere, Veron, Philippe, Leborgne, Christian, Deschamps, Jack-Yves, Moullec, Sophie, Fromes, Yves, Collaud, Fanny, Boutin, Sylvie, Latournerie, Virginie, van Wittenberghe, Laetitia, Delache, Benoit, Le Grand, Roger, Dereuddre-Bosquet, Nathalie, Benveniste, Olivier, Moullier, Philippe, Masurier, Carole, Merten, Otto, Mingozzi, Federico (2020) Capsid-specific removal of circulating antibodies to adeno-associated virus vectors. Scientific Reports, 10. doi:10.1038/s41598-020-57893-z |
| Not Yet Imported: - journal-article : 10.1038/mt.2013.244 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.omtm.2024.101195 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.semnephrol.2017.05.020 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1056/NEJMoa1612567 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: Nature Medicine - journal-article : 10.1038/s41591-020-0911-7 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Cao (2025) Gene Ther. Safety, Efficacy, and Immunogenicity of a Novel IgG Degrading Enzyme (KJ103): Results from Two Randomised, Blinded, Phase 1 Clinical Trials 32, 223 | |
| Not Yet Imported: - journal-article : 10.1186/s42826-024-00195-6 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.theriogenology.2007.09.030 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
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| Not Yet Imported: Cold Spring Harbor Perspectives in Biology - journal-article : 10.1101/cshperspect.a003566 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: Nature Biotechnology - journal-article : 10.1038/s41587-019-0357-y If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: eLife - journal-article : 10.7554/eLife.18858 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1038/s41587-023-01989-0 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1002/jmv.29305 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use ZOLGENSMA Safely and Effectively. See Full Prescribing Information for ZOLGENSMA. ZOLGENSMA® (Onasemnogene Abeparvovec-Xioi) Suspension, for Intravenous Infusion. Initial U.S. Approval: 2019, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=68cd4f06-70e1-40d8-bedb-609ec0afa471. | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use LUXTURNA Safely and Effectively. See Full Prescribing Information for LUXTURNA.LUXTURNA (Voretigene Neparvovec-Rzyl) Intraocular Suspension for Subretinal Injection. Initial U.S. Approval: 2017, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=08313a24-e7ce-457a-bb3f-161bc45517ee. | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use HEMGENIX Safely and Effectively. See Full Prescribing Information for HEMGENIX. HEMGENIX (Etranacogene Dezaparvovec-Drlb) Suspension, for Intravenous Infusion. Initial U.S. Approval: 2022, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=35b2db65-4c6c-4173-ab56-b2bca69193bd&type=display. | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use ROCTAVIAN Safely and Effectively. See Full Prescribing Information for ROCTAVIAN. ROCTAVIAN (Valoctocogene Roxaparvovec-Rvox) Suspension for Intravenous Infusion. Initial U.S. Approval: 2023, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=0dcf7185-8e1c-456a-9d4e-7cc316400118. | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use ELEVIDYS® Safely and Effectively. See Full Prescribing Information for ELEVIDYS. ELEVIDYS (Delandistrogene Moxeparvovec-Rokl) Suspension, for Intravenous Infusion. Initial U.S. Approval: 2023, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=3525abcd-fbd2-46c5-9706-cffdf2e8a361&type=display. | |
| (2026, March 26). These Highlights Do Not Include All the Information Needed to Use KEBILIDI Safely and Effectively. See Full Prescribing Information for KEBILIDI. KEBILIDI (Eladocagene Exuparvovec-Tneq) Suspension, for Intraputaminal Infusion. Initial U.S. Approval: 2024, Available online: https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=9d6a6401-c6b5-4f29-af11-67707d249482&type=display. | |
| European Medicines Agency (EMA) (2026, March 26). Upstaza. Available online: https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza. | |
| Not Yet Imported: - journal-article : 10.1186/s13075-022-02886-8 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Mok (2024) Proc. Natl. Acad. Sci. USA Post-Immunotherapy CTLA-4 Ig Treatment Improves Antitumor Efficacy 121, e2404661121 | |
| Not Yet Imported: - journal-article : 10.1111/jth.14757 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
![]() | Meliani, Amine, Boisgerault, Florence, Hardet, Romain, Marmier, Solenne, Collaud, Fanny, Ronzitti, Giuseppe, Leborgne, Christian, Costa Verdera, Helena, Simon Sola, Marcelo, Charles, Severine, Vignaud, Alban, van Wittenberghe, Laetitia, Manni, Giorgia, Christophe, Olivier, Fallarino, Francesca, Roy, Christopher, Michaud, Alicia, Ilyinskii, Petr, Kishimoto, Takashi Kei, Mingozzi, Federico (2018) Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. Nature Communications, 9. doi:10.1038/s41467-018-06621-3 |
| Not Yet Imported: Molecular Therapy - Methods & Clinical Development - journal-article : 10.1016/j.omtm.2021.06.015 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
![]() | Fuchs, Sebastian P.; Mondragon, Paula G.; Zabizhin, Rachel; Tomer, Shallu; Wang, Li; Cook, Ethan; Dudley, Dawn M.; Weisgrau, Kimberly L.; Furlott, Jessica; Coonen, Jennifer; et al. (2025) Transient rapamycin treatment avoids unwanted host immune responses toward AAV-delivered anti-HIV antibodies. Nature Communications, 16 (1). doi:10.1038/s41467-025-63970-6 |
![]() | Ilyinskii, Petr O., Michaud, Alicia M., Roy, Christopher J., Rizzo, Gina L., Elkins, Stephanie L., Capela, Teresa, Chowdhury, Aparajita C., Leung, Sheldon S., Kishimoto, Takashi K. (2021) Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles. Science Advances, 7 (9). doi:10.1126/sciadv.abd0321 |
| Not Yet Imported: - journal-article : 10.1089/hum.2023.194 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1097/TP.0000000000004689 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: Transplantation - journal-article : 10.1097/TP.0000000000003496 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1172/jci.insight.139881 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Smith (2024) Mol. Ther. Engineered IgM and IgG Cleaving Enzymes for Mitigating Antibody Neutralization and Complement Activation in AAV Gene Transfer 32, 2080 | |
| Not Yet Imported: - journal-article : 10.1016/j.ymthe.2005.03.018 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1016/j.cellimm.2017.12.002 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.3389/fimmu.2020.00618 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.3389/fimmu.2020.00494 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - book-chapter : 10.1007/978-1-0716-2707-5_18 If you would like this item imported into the Digital Library, please contact us quoting Book ID 9781071627068 | |
| Not Yet Imported: Molecular Therapy - journal-article : 10.1038/mt.2011.108 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: American Journal of Transplantation - journal-article : 10.1111/ajt.16828 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1038/mt.2015.179 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1038/mtm.2013.9 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Cheng (2025) Mol. Ther. Methods Clin. Dev. Probing Aspects of Extracellular Vesicle Associated AAV Allows Increased Vector Yield and Insight into Its Transduction and Immune-Evasive Properties 33, 101407 | |
| Not Yet Imported: Blood Advances - journal-article : 10.1182/bloodadvances.2017010181 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Not Yet Imported: - journal-article : 10.1038/s41593-021-00969-4 If you would like this item imported into the Digital Library, please contact us quoting Journal ID | |
| Wang (2024) Interdiscip. Med. Adeno-associated Virus Vectors for Gene Therapy—Focusing on Melanoma 2, e20240031 |
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