COMPOSITIONS AND METHODS USEFUL FOR NON-INVASIVE DELIVERY OF THERAPEUTIC MOLECULES TO THE BLOODSTREAM

摘要

A non-invasive method for obtaining pharmaceutically effective levels of protein in the bloodstream by administering rAAV containing a transgene encoding the secretable or extracellular membrane-bound protein via inhalation, is provided. Suitably, the transgene product is under the control of a lung-specific promoter. Also provided are pharmaceutical kits containing rAAV encoding the secretable protein in a suspension suitable for delivery via intranasal or oral inhalation.