Complement System Response to Adeno-Associated Virus Vector Gene Therapy
Open accessAbstract
Adeno-associated virus (AAV) vectors represent a novel tool for the delivery of genetic therapeutics and enable the treatment of a wide range of diseases. Success of this new modality is challenged, however, by cases of immune-related toxicities that complicate the clinical management of patients and potentially limit the therapeutic efficacy of AAV gene therapy. While significant progress has been made to manage immune-related liver enzyme elevations following systemic AAV delivery in humans, recent clinical trials utilizing high vector doses have highlighted a new challenge to AAV gene transfer—activation of the complement system. While current in vitro models implicate AAV-specific antibodies in the initiation of the classical complement pathway, evidence from in vivo pre-clinical and clinical studies suggests that the alternative pathway also contributes to complement activation. A convergence of AAV-specific, environmental, and patient-specific factors shaping complement responses likely contributes to differential outcomes seen in clinical trials, from priming of the adaptive immune system to serious adverse events such as hepatotoxicity and thrombotic microangiopathy. Research focused on the interplay of patient-specific and AAV-related factors driving complement activation is needed to understand and identify critical components in the complement cascade to target and devise strategies to mitigate vector-related immune responses.
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Complement activation in response to systemic AAV administration and related acute toxicities has been reported for numerous clinical and preclinical studies.
“Complement activation in response to systemic AAV administration and related acute toxicities has been reported for numerous clinical and preclinical studies (39, 52–56).”
From Intravitreal AAV vector delivery induces integrin-dependent ocular inflammation, complement activation, and antiviral and DNA damage responses (2026) · cites this paper as [56]
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- Authors
- Kropf, E., Markusic, D. M., Majowicz, A., Mingozzi, F., & Kuranda, K.
- Affiliations
- Spark Therapeutics (United States) (US)
- Corresponding author
- Kuranda, Klaudia
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2024-07
- Volume
- 35
- Issue
- 13-14
- Pages
- 425-438
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W4391103529
- Cited by
- 39
- References
- 57
- Citations by year
- 2026: 21 · 2025: 14 · 2024: 4
- FWCI
- 10.36
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Human Gene Therapy (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Immunology and Microbiology › Immunology › Complement system in diseases
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- adeno-associated virus vectors, gene therapy, complement activation, alternative complement pathway, classical complement pathway, immune-related toxicity, hepatotoxicity, thrombotic microangiopathy, vector dose, immune response to gene therapy
- MeSH terms
- Dependovirus, Animals, Complement System Proteins, Complement Activation, Genetic Vectors, Humans, Genetic Therapy, Gene Transfer Techniques
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