AAV vectors for in vivo gene therapy of rheumatoid arthritis

摘要

Disclosed is a method for delivering a nucleic acid molecule to a rheumatoid synovial cells in vivo, the method comprising the steps of (a) providing a recombinant AAV virion (rAAV) comprising capsid proteins of AAV serotype 5 or AAV serotype 2, wherein the rAAV virion comprises a rAAVX vector, the rAAVX vector comprising an expression element operably linked to a nucleic acid sequence; and, (b) bringing the rAAV virion into contact with the synovial cell, whereby transduction of the rAAVX vector results in expression of the nucleic acid sequence in the transduced synovial cells.