AAV-shRNA PTH1R

AAV-shRNA PTH1R is an experimental gene therapy designed to treat pulmonary hypertension (PH) by knocking down the expression of the parathyroid hormone receptor 1 (PTH1R) in the lungs. Delivered via inhalation using an adeno-associated virus (AAV) vector, the therapy utilizes short hairpin RNA (shRNA) to silence PTH1R signaling. Research indicates that parathyroid hormone (PTH) exacerbates pulmonary hypertension by promoting the migration and proliferation of pulmonary artery smooth muscle cells (PASMCs) through PTH1R-ERK signaling. In preclinical models, local knockdown of PTH1R in the lungs significantly improved pulmonary hemodynamics and reduced right ventricular hypertrophy, suggesting that the PTH/PTH1R axis is a viable therapeutic target for PH.