What question did this study set out to answer?

This review aims to assess the safety concerns of AAV vectors in gene therapy by analyzing clinical data on toxicity mechanisms and mitigation strategies.

May 29, 2026International Journal of Molecular SciencesOpen Access

Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization

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Authors

TTTatiana S. TsaregorodtsevaKazan Federal University MRMaria A. RadyukhinaKazan Federal University AAAisylu I. AyupovaKazan Federal University

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Review analyzes AAV gene therapy toxicity and clinical risks, suggesting strategies for safety improvement.

Key Points

This review aims to assess the safety concerns of AAV vectors in gene therapy by analyzing clinical data on toxicity mechanisms and mitigation strategies.
Systematic analysis of clinical data regarding AAV vector administration
Examination of dose-dependent toxicity and immune responses
Evaluation of contemporary strategies for minimizing risks in gene therapy
Identified a correlation between systemic delivery efficacy and dose-dependent toxicity.
Mechanisms of toxicity include immune responses and organ-specific burdens, particularly hepatic effects.
Evolving strategies for toxicity mitigation include immunosuppressive therapies and vector engineering techniques.

Cite This Study

Tsaregorodtseva et al. (2026) studied this question.

synapsesocial.com/papers/6a192dbbfab5b468c44169ae https://doi.org/10.3390/ijms27114818

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Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization

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Cite This Study

Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization

Discussion

Member takes

Implication

Key Points

Cite This Study

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