C-reactive protein (CRP) is essential for efficient systemic transduction of recombinant adeno-associated virus vector 1 (rAAV-1) and rAAV-6 in mice
Open accessAbstract
The clinical relevance of gene therapy using the recombinant adeno-associated virus (rAAV) vectors often requires widespread distribution of the vector, and in this case, systemic delivery is the optimal route of administration. Humoral blood factors, such as antibodies or complement, are the first barriers met by the vectors administered systemically. We have found that other blood proteins, galectin 3 binding protein (G3BP) and C-reactive protein (CRP), can interact with different AAV serotypes in a species-specific manner. While interactions of rAAV vectors with G3BP, antibodies, or complement lead to a decrease in vector efficacy, systemic transduction of the CRP-deficient mouse and its respective control clearly established that binding to mouse CRP (mCRP) boosts rAAV vector 1 (rAAV-1) and rAAV-6 transduction efficiency in skeletal muscles over 10 times. Notably, the high efficacy of rAAV-6 in CRP-deficient mice can be restored by reconstitution of the CRP-deficient mouse with mCRP. Human CRP (hCRP) does not interact with either rAAV-1 or rAAV-6, and, consequently, the high efficiency of mCRP-mediated muscle transduction by these serotypes in mice cannot be translated to humans. No interaction of mCRP or hCRP was observed with rAAV-8 and rAAV-9. We show, for the first time, that serum components can significantly enhance rAAV-mediated tissue transduction in a serotype- and species-specific manner. Bioprocessing in body fluids should be considered when transfer of a preclinical proof of concept for AAV-based gene therapy to humans is planned.
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Triggering a systemic immune response against an AAV-based vector can result in unintended side effects such as transgene expression in lymphoid organs or systemic complement activation.
“triggering a systemic immune response against an AAV-based vector can result in unintended side effects such as transgene expression in lymphoid organs [263,264] or systemic complement activation [265,266].”
From Bruch’s Membrane: A Key Consideration with Complement-Based Therapies for Age-Related Macular Degeneration (2023) · cites this paper as [266]
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- Authors
- Denard, J., Marolleau, B., Jenny, C., Rao, T. N., Fehling, H. J., Voit, T., & Svinartchouk, F.
- Affiliations
- Genethon, Evry, France
- Genethon (France) (FR)
- Joslin Diabetes Center (US)
- Universität Ulm (DE)
- Université Pierre et Marie Curie, UM76, Inserm U974, CNRS, UMR7215, Institut de Myologie, Paris, France
- Venue
- Journal of Virology
- Publisher
- American Society for Microbiology
- Published
- 2013-10
- Volume
- 87
- Issue
- 19
- Pages
- 10784-10791
- ISSN
- 0022-538X (ISSN-L)
- 1098-5514
- PMC
- PMC3807418
- OpenAlex
- W2142763900
- Cited by
- 31
- References
- 56
- Citations by year
- 2024: 1 · 2023: 4 · 2022: 4 · 2021: 5 · 2020: 8 · 2019: 1 · 2018: 2 · 2016: 3 · 2015: 1 · 2014: 1 · 2013: 1
- FWCI
- 1.33
- Type
- journal article
- Language
- en
- Open access
- green
- europepmc.org
- Locations
- Journal of Virology (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- HAL (submittedVersion)
- Funders
- Institut Gustave-Roussy
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Keywords
- C-reactive protein, recombinant adeno-associated virus, AAV serotypes, systemic gene therapy, LGALS3BP, serum proteins, AAV8, AAV9, preclinical to clinical translation
- MeSH terms
- Dependovirus, Animals, C-Reactive Protein, Genetic Vectors, Humans, Luciferases, Male, Mice, Inbred C57BL, RNA, Messenger, Transduction, Genetic, Blotting, Western, Mice, Knockout, Muscle, Skeletal, Reverse Transcriptase Polymerase Chain Reaction, Immunoprecipitation, Mice, Real-Time Polymerase Chain Reaction
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 ›
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