Gene therapy for alpha-1 antitrypsin deficiency

Abstract excerpt

Alpha-1 antitrypsin (AAT) deficiency is a common single-gene disorder among Northern Europeans and North Americans. The carrier frequency for the common missense mutation (Z-AAT) ranges from 4% in the US to nearly 25% in the Republic of Ireland. Severe AAT deficiency (plasma levels below 11 μm) is most commonly associated with an adult-onset lung disease, with pan-acinar emphysema and airway inflammation, which...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to RNA Interference and Gene Delivery
• Share to Viral Infections and Immunology Research
• Share to Virus-based gene therapy research