Direct gene delivery strategies for the treatment of rheumatoid arthritis

Steven C. Ghivizzani, Thomas J. Oligino, Joseph C. Glorioso, Paul D. Robbins, Christopher H. Evans

Research output: Contribution to journalReview articlepeer-review

53 Scopus citations


Gene therapy offers a novel and innovative approach to the delivery of therapeutic proteins to the joints of patients with arthritis. Several viral vectors, including adenovirus, adeno-associated virus, retrovirus and herpes simplex virus, are capable of delivering exogenous cDNAs to the synovial lining, enabling effective levels of intra-articular transgene expression following direct injection to the joint. The expression of certain gene products has proven to be sufficient to inhibit the progression of disease in animals with experimental arthritis. Non-viral methods of gene transfer, however, are less satisfactory, and are limited by toxicity and transience of expression. Although the principle of direct gene delivery to the joint has been demonstrated, maintaining persistent intra-articular transgene expression remains a challenge.

Original languageEnglish (US)
Pages (from-to)259-267
Number of pages9
JournalDrug Discovery Today
Issue number5
StatePublished - Mar 1 2001
Externally publishedYes


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