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Gene Therapy for Rheumatoid Arthritis

Current Status and Future Prospects

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Abstract

Rheumatoid arthritis (RA) was one of the earliest targets for gene therapy. Since the first clinical trial involving gene therapy in RA was initiated in 1996, eight clinical trials have been conducted assessing gene therapy in RA. Gene therapy has benefited from advances in biologics in terms of the increasing choice of novel, efficient targets to treat RA and also from the optimization of the delivery systems. Several strategies are possible; one of particular interest is local gene therapy directed to rheumatic joints, which avoids systemic vector diffusion. In this review, we discuss (i) gene therapeutic approaches that have been attempted for patients with RA, and (ii) novel strategies that are in development for delivery into patients. We analyze the advantages and disadvantages of the various approaches and how best to optimize them with regard to choosing the most promising vectors and strategies to allow for efficient, long-term, safe delivery of gene therapy in RA.

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Acknowledgments

No sources of funding were used to assist in the preparation of this review. Florence Apparailly has received grants and patents from Arthrogen BV. Sylvie Fabre has no conflicts of interest that are directly relevant to the content of this review.

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Authors and Affiliations

  1. Inserm, U 844, 80 rue Augustin Fliche, BP 74103, 34295, Montpellier cedex 5, France

    Sylvie Fabre & Florence Apparailly

  2. Université Montpellier 1, UFR de Médecine, Montpellier, France

    Sylvie Fabre & Florence Apparailly

  3. CHU Lapeyronie, Service Clinique d’Immuno-Rhumatologie, Montpellier, France

    Sylvie Fabre & Florence Apparailly

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  1. Sylvie Fabre
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  2. Florence Apparailly
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Correspondence to Sylvie Fabre.

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Fabre, S., Apparailly, F. Gene Therapy for Rheumatoid Arthritis. BioDrugs 25, 381–391 (2011). https://doi.org/10.2165/11595490-000000000-00000

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