Gene therapy represents a promising strategy for treating the pulmonary manifestations of Alpha-1 antitrypsin deficiency by potentially providing sustained therapeutic AAT levels after a single administration.
Should not change AAT infusion practice; leaves open clinical translation of preclinical gene therapy vectors.
Alpha-1 antitrypsin (AAT) deficiency, characterized by low plasma levels of the serine protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of the lung from neutrophil proteases. Although AAT augmentation therapy with purified AAT protein is efficacious, it requires weekly to monthly intravenous infusion of AAT purified from pooled human plasma, has the risk of viral contamination and allergic reactions, and is costly. As an alternative, gene therapy offers the advantage of single administration, eliminating the burden of protein infusion, and reduced risks and costs. The focus of this review is to describe the various strategies for AAT gene therapy for the pulmonary manifestations of AAT deficiency and the state of the art in bringing AAT gene therapy to the bedside.
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Chiuchiolo et al. (2016) studied this question.
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