Safety and long-term efficacy of AAV1.SERCA2a using nebulizer delivery in a pig model of pulmonary hypertension

Pulm Circ. 2018 Oct-Dec;8(4):2045894018799738. doi: 10.1177/2045894018799738. Epub 2018 Aug 21.

Abstract

Nebulization delivery of adeno-associated virus serotype 1 encoding sarcoplasmic reticulum Ca2+-ATPase2a (AAV1.SERCA2a) gene was examined in a Yukatan miniature swine model of chronic pulmonary hypertension (n = 13). Nebulization of AAV1.SERCA2a resulted in homogenous distribution of vectors, lower pulmonary vascular resistance, and a trend towards better long-term survival compared to control animals.

Keywords: airway delivery; gene therapy; nebulization; post-capillary pulmonary hypertension.