Key result
Gene transfer of therapeutic proteins like IL-1ra to the synovium demonstrates feasibility and efficacy in animal models, leading to the approval of the first human gene therapy trial for arthritis.
Population
Animal models of arthritis and patients with rheumatoid arthritis scheduled for joint replacement
Design
Review
Authors
Discussion
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Member takes
Implication
Supports first human gene therapy trials in arthritis; leaves open proof of clinical efficacy and safety.
Structured PICO
P
Population
Animal models of arthritis (rabbit knee, mouse) and patients with rheumatoid arthritis scheduled for joint replacement (upcoming clinical trial)
I
Intervention
Gene therapy delivering therapeutic proteins (e.g., IL-1 receptor antagonist protein [IL-1ra/IRAP]) via viral or nonviral vectors
Preclinical evidence demonstrates the feasibility and efficacy of treating arthritis by gene transfer of IL-1ra, leading to the approval of the first human gene therapy trial for rheumatoid arthritis.
Limitations
- Adenovirus and herpes virus vectors are not completely defective, may induce an immune response, and provide only transient expression.
- Difficulty in generating high titer recombinant AAV virus.
- In vivo gene delivery using cationic liposomes has met with only marginal success.
Cite This Study
Robbins et al. (1994) conducted a review in Arthritis (including rheumatoid arthritis). Gene therapy (e.g., IL-1ra gene transfer) was evaluated. Gene transfer of therapeutic proteins like IL-1ra to the synovium demonstrates feasibility and efficacy in animal models, leading to the approval of the first human gene therapy trial for arthritis.