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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

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Reference TypeJournal (article/letter/editorial)
TitleSafety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization
JournalInternational Journal of Molecular Sciences
AuthorsTsaregorodtseva, 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
Year2026 (May 27)Volume27
Issue11
PublisherMDPI AG
DOIdoi:10.3390/ijms27114818Search in ResearchGate
Generate Citation Formats
Mindat Ref. ID20032749Long-form Identifiermindat:1:5:20032749:9
GUID0
Full ReferenceTsaregorodtseva, 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 TextTsaregorodtseva, 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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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: Molecular Therapy - Methods & Clinical Development - journal-article : 10.1016/j.omtm.2023.02.015

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Not Yet Imported: - journal-article : 10.47102/annals-acadmedsg.V37N7p591

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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

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Not Yet Imported: The Journal of Gene Medicine - journal-article : 10.1002/jgm.1161

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Not Yet Imported: - journal-article : 10.1089/hum.2014.082

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Not Yet Imported: - journal-article : 10.1089/hum.2009.157

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Not Yet Imported: Gene Therapy - journal-article : 10.1038/gt.2017.75

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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
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(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.
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Mok (2024) Proc. Natl. Acad. Sci. USA Post-Immunotherapy CTLA-4 Ig Treatment Improves Antitumor Efficacy 121, e2404661121
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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

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Not Yet Imported: Molecular Therapy - journal-article : 10.1038/mt.2011.108

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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

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Wang (2024) Interdiscip. Med. Adeno-associated Virus Vectors for Gene Therapy—Focusing on Melanoma 2, e20240031


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