Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients
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Pre-existing and post-administration anti-AAV immunity can severely inhibit systemic AAV vector gene delivery.
“However, another challenge to AAV-mediated gene therapy is humoral, anti-AAV antibody mediated immunity, which results from childhood exposure to one or more serotypes or from prior administration of AAV vectors and poses a significant challenge to AAV gene therapy.26,27”
From Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates (2015) · cites this paper as [27]
The trial report (page unavailable in our copy) observed antibodies against AAV1 capsid proteins after intramuscular vector administration and says the later loss of efficacy was potentially related to an immune response. It does not address pre-existing immunity from childhood exposure or prior AAV treatment, nor show severe inhibition of systemic vector delivery. (LLM, conf=0.86)
Experimental — accuracy not yet measuredAAV has been successful in clinical gene therapy trials for LCA, hemophilia, familial lipoprotein lipase deficiency, and choroideremia.
“Adeno-associated virus has been successful in clinical gene therapy trials for Leber's congenital amaurosis, hemophilia, familial lipoprotein lipase deficiency, and choroideremia.8–15,26,27,60,61”
From Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates (2015) · cites this paper as [27]
The clinical-trial report describes an AAV1 gene-therapy trial in eight lipoprotein lipase–deficient patients and a significant reduction in triglycerides at 12 weeks. But its results passage says only four patients reached the primary efficacy endpoint and efficacy was lost at 18–31 months, so calling the trial simply “successful” overstates its findings. This source addresses the lipoprotein lipase deficiency part of the multi-citation sentence. (LLM, conf=0.84)
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References 4 · Cited by 209
- Authors
- Stroes, E. S., Nierman, M. C., Meulenberg, J. J., Franssen, R., Twisk, J., Henny, C. P., Maas, M. M., Zwinderman, A. H., Ross, C., Aronica, E., High, K. A., Levi, M. M., Hayden, M. R., Kastelein, J. J., & Kuivenhoven, J. A.
- Affiliations
- University of British Columbia (CA)
- ORCID
- Venue
- Arteriosclerosis, Thrombosis, and Vascular Biology
- Published
- 2008-12
- Volume
- 28
- Issue
- 12
- Pages
- 2303-2304
- ISSN
- 1079-5642 (ISSN-L)
- 1524-4636
- OpenAlex
- W2145127625
- Cited by
- 209
- References
- 4
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 1 · 2023: 7 · 2022: 4 · 2021: 10 · 2020: 4 · 2019: 12 · 2018: 14 · 2017: 10 · 2016: 13 · 2015: 26 · 2014: 17 · 2013: 17 · 2012: 19
- FWCI
- 11.32
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Arteriosclerosis Thrombosis and Vascular Biology (publishedVersion)
- PubMed (publishedVersion)
- Pure Amsterdam UMC (publishedVersion)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- University of Groningen research database (University of Groningen / Centre for Information Technology) (submittedVersion)
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipid metabolism and disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Surgery › Lipoproteins and Cardiovascular Health
- Keywords
- lipoprotein lipase, triglyceride lowering, lipoprotein lipase deficiency, hypertriglyceridemia, gene replacement therapy
- MeSH terms
- Dependovirus, Genetic Vectors, Humans, Injections, Intramuscular, Lipoprotein Lipase, Hyperlipoproteinemia Type I, Time Factors, Triglycerides, Genetic Therapy
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