recombinant adeno-associated virus expressing microRNA-39

Recombinant adeno-associated virus expressing microRNA-39 is a gene therapy candidate developed by researchers at the University of Massachusetts Chan Medical School for the treatment of osteoporosis and inflammatory arthritis. The therapy utilizes a bone-targeted recombinant adeno-associated viral (rAAV) vector to deliver and overexpress microRNA-39 (miR-39). Mechanistically, miR-39 acts as a regulator of the Schnurri-3 (SHN3) signaling pathway by repressing the expression of Tumor Necrosis Factor alpha (TNFα). This repression leads to the SHN3-dependent activation of WNT signaling, which robustly enhances osteoblast-mediated bone formation. Preclinical studies in mouse models have demonstrated that this approach can reverse bone loss in postmenopausal and senile osteoporosis, as well as prevent inflammation-induced bone loss in models of rheumatoid arthritis, without detectable adverse effects in non-skeletal tissues.

Other names

rAAV-miR-39rAAV-miR39rAAV-miR 39rAAV-microRNA-39rAAV-microRNA39rAAV-microRNA 39bone-targeted rAAV-miR-39