AAV-shPiezo2

AAV-shPiezo2 is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver short hairpin RNA (shRNA) to silence the expression of the mechanosensitive ion channel Piezo2. Developed by academic institutions including Sun Yat-Sen University and Huazhong University of Science and Technology, the therapeutic candidate is being investigated in preclinical models for the treatment of rheumatoid arthritis (RA) and discogenic low back pain. In RA models, local intra-articular injection of AAV-shPiezo2 silences Piezo2 in fibroblast-like synoviocytes, reducing joint inflammation, cartilage/bone destruction, and osteoclast activation by disrupting the vesicular trafficking and secretion of RANKL. In models of discogenic low back pain, AAV-shPiezo2-mediated silencing of Piezo2 in the dorsal root ganglion alleviates pain-related behaviors, suppresses inflammation, and delays cartilage endplate degeneration.