Trial reportHuman gene therapy2009
Clinical responses to gene therapy in joints of two subjects with rheumatoid arthritis.
Trial report in Human gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
33 citing papers in PubMed, 82 citations in OpenAlex.
- Engineered self-regulating macrophages for targeted anti-inflammatory drug delivery.Arthritis research & therapy · 2024Article
- Gene therapy for chronic pain management.Cell reports. Medicine · 2024Review
- Engineered Self-Regulating Macrophages for Targeted Anti-inflammatory Drug Delivery.Research square · 2024Article
- Gene Therapy for Regenerative Medicine.Pharmaceutics · 2023Review
- A first report on the efficacy of a single intra-articular administration of blood cell secretome, triamcinolone acetonide, and the combination of both in dogs with osteoarthritis.BMC veterinary research · 2022Article
- Synthetic gene circuits for preventing disruption of the circadian clock due to interleukin-1-induced inflammation.Science advances · 2022Article
- Intra-articular injection of placental mesenchymal stromal cells ameliorates pain and cartilage anabolism/catabolism in knee osteoarthritis.Frontiers in pharmacology · 2022Article
- Immunoengineering the next generation of arthritis therapies.Acta biomaterialia · 2021Review
- Orthopaedic Gene Therapy: Twenty-Five Years On.JBJS reviews · 2021Article
- A Synthetic Gene Circuit for Self-Regulating Delivery of Biologic Drugs in Engineered Tissues.Tissue engineering. Part A · 2019Article
- Biomaterial-guided delivery of gene vectors for targeted articular cartilage repair.Nature reviews. Rheumatology · 2019Review
- Editorial: Arthritis Gene Therapy Using Interleukin-1 Receptor Antagonist.Arthritis & rheumatology (Hoboken, N.J.) · 2018Article
- Bone erosions in rheumatoid arthritis: recent developments in pathogenesis and therapeutic implications.Journal of musculoskeletal & neuronal interactions · 2018Review
- Gene Delivery to Joints by Intra-Articular Injection.Human gene therapy · 2018Review
- Soluble interleukin 23 receptor gene therapy with adeno-associated vectors for the treatment of multiple sclerosis.Neural regeneration research · 2017Article
- Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model.Molecular therapy. Methods & clinical development · 2016Article
- Gene Therapy for Autoimmune Disease.Clinical reviews in allergy & immunology · 2015Review
- scAAVIL-1ra dosing trial in a large animal model and validation of long-term expression with repeat administration for osteoarthritis therapy.Gene therapy · 2015Article
- Cytokine-modulating strategies and newer cytokine targets for arthritis therapy.International journal of molecular sciences · 2014Review
- Using genes to facilitate the endogenous repair and regeneration of orthopaedic tissues.International orthopaedics · 2014Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 1 institution in 1 country.
Funding
Abstract
This paper provides the first evidence of a clinical response to gene therapy in human arthritis. Two subjects with rheumatoid arthritis received ex vivo, intraarticular delivery of human interleukin-1 receptor antagonist (IL-1Ra) cDNA. To achieve this, autologous synovial fibroblasts were transduced with a retrovirus, MFG-IRAP, carrying IL-1Ra as the transgene, or remained as untransduced controls. Symptomatic metacarpophalangeal (MCP) joints were injected with control or transduced cells. Joints were clinically evaluated on the basis of pain; the circumference of MCP joint 1 was also measured. After 4 weeks, joints underwent surgical synovectomy. There were no adverse events in either subject. The first subject responded dramatically to gene transfer, with a marked and rapid reduction in pain and swelling that lasted for the entire 4 weeks of the study. Remarkably, joints receiving IL-1Ra cDNA were protected from flares that occurred during the study period. Analysis of RNA recovered after synovectomy revealed enhanced expression of IL-1Ra and reduced expression of matrix metalloproteinase-3 and IL-1beta. The second subject also responded with reduced pain and swelling. Thus, gene transfer to human, rheumatoid joints can be accomplished safely to produce clinical benefit, at least in the short term. Using this ex vivo procedure, the transgene persisted within the joint for at least 1 month. Further clinical studies are warranted.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.