ArticlePloS one2015
Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.
Article in PloS one, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 31 citations in OpenAlex.
- Role of Gut Microbiota and Metabolite Remodeling on the Development and Management of Rheumatoid Arthritis: A Narrative Review.Veterinary sciences · 2025Review
- Current Immunotherapy Strategies for Rheumatoid Arthritis: The Immunoengineering and Delivery Systems.Research (Washington, D.C.) · 2023Review
- Immunoengineering the next generation of arthritis therapies.Acta biomaterialia · 2021Review
- Candidates for Intra-Articular Administration Therapeutics and Therapies of Osteoarthritis.International journal of molecular sciences · 2021Review
- Liposomal Nanosystems in Rheumatoid Arthritis.Pharmaceutics · 2021Review
- Gene delivery for immunoengineering.Current opinion in biotechnology · 2020Review
- Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates.Human gene therapy · 2020Article
- Gene therapy for chondral and osteochondral regeneration: is the future now?Cellular and molecular life sciences : CMLS · 2018Review
- Gene Delivery to Joints by Intra-Articular Injection.Human gene therapy · 2018Review
- Histone deacetylase 6 inhibition reduces cysts by decreasing cAMP and CaThe Journal of biological chemistry · 2017Article
- Regenerative medicine: Breaking Prometheus's curse for cartilage regeneration.Nature reviews. Rheumatology · 2017Article
- Pregnancy-induced gene expression changes in vivo among women with rheumatoid arthritis: a pilot study.Arthritis research & therapy · 2017Article
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Authors and funding
8 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionProof of concept for local gene therapy for the treatment of arthritis with immunomodulatory cytokine interferon beta (IFN-β) has shown promising results in animal models of rheumatoid arthritis (RA). For the treatment of RA patients, we engineered a recombinant adeno-associated serotype 5 vector (rAAV5) encoding human (h)IFN-β under control of a nuclear factor κB promoter (ART-I02).
methodsThe potency of ART-I02 in vitro as well as biodistribution in vivo in arthritic animals was evaluated to characterize the vector prior to clinical application. ART-I02 expression and bioactivity after transduction was evaluated in fibroblast-like synoviocytes (FLS) from different species. Biodistribution of the vector after local injection was assessed in a rat adjuvant arthritis model through qPCR analysis of vector DNA. In vivo imaging was used to investigate transgene expression and kinetics in a mouse collagen induced arthritis model.
resultsTransduction of RA FLS in vitro with ART-I02 resulted in high expression levels of bioactive hIFN-β. Transduction of FLS from rhesus monkeys, rodents and rabbits with ART-I02 showed high transgene expression, and hIFN-β proved bioactive in FLS from rhesus monkeys. Transgene expression and bioactivity in RA FLS were unaltered in the presence of methotrexate. In vivo, vector biodistribution analysis in rats after intra-articular injection of ART-I02 demonstrated that the majority of vector DNA remained in the joint (>93%). In vivo imaging in mice confirmed local expression of rAAV5 in the knee joint region and demonstrated rapid detectable and sustained expression up until 7 weeks.
conclusionsThese data show that hIFN-β produced by RA FLS transduced with ART-I02 is bioactive and that intra-articular delivery of rAAV5 drives expression of a therapeutic transgene in the joint, with only limited biodistribution of vector DNA to other tissues, supporting progress towards a phase 1 clinical trial for the local treatment of arthritis in patients with RA.
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