Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease

• DOI: 10.15326/jcopdf.5.4.2017.0160
• Publication Date: 2018-10-02T23:57:50Z
• Authors: Katie M. Stiles; Dolan Sondhi; Stephen M. Kaminsky; Bishnu P. De; Jonathan B. Rosen...; Ronald G. Crystal
• Keywords: Alpha 1-antitrypsin deficiency; Alpha (finance)

Alpha-1 antitrypsin deficiency (AATD) manifests primarily as early-onset emphysema caused by the destruction of lung neutrophil elastase due to low amounts serine protease inhibitor alpha-1 (AAT). The current therapy involves weekly intravenous infusions AAT-derived from pooled human plasma that is efficacious, yet costly. Gene applications designed provide constant levels AAT protein are currently under development. challenge for gene sufficient normalize level and anti-neutrophil capacity in lung. One strategy administration an adeno-associated virus (AAV) vector pleural space providing both local systemic production reach consistent therapeutic levels. This review focuses on strategy, advantages, challenges, updates intrapleural vectors treatment AATD.