Engineered AAVs for Enhanced Transduction of Submucosal Cells within the Lung Following Intravenous Delivery

Abstract

Lung diseases are one of the leading causes of morbidity and mortality worldwide, highlighting the need for vectors capable of efficiently transducing cells within the lung. Recent developments in gene delivery have demonstrated that the use of gene therapies to treat disease is a realistic and achievable goal. Adeno-associated viruses (AAVs) have quickly become the vector of choice in both laboratory and clinical settings due to their strong safety record and stable expression in vivo, especially when compared to other viruses. However, the naturally occurring AAVs tend to have severely limited tropisms in lung tissues, as expression following intranasal delivery is generally inefficient and restricted to the airway epithelium. One solution to this obstacle is to utilize a systemically injected AAV in conjunction with inhalant based delivery to cover a broad population of cells throughout the lung.

Additional details