Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients

Alpha-1 antitrypsin deficiency (AATD) is an autosomal co-dominant disease which predisposes patients to early-onset emphysema and liver cirrhosis.1 Estimates of the number of adult patients with AATD exhibiting clinically significant liver disease have varied from 10% (defined by aminotransferase abnormalities)2,3 to 18% in a UK cohort assessed by ultrasound (+/ − biopsy)4 to approximately 35% in those with an Ishak fibrosis score ≥2 assessed histologically after liver biopsy.5 A Swedish autopsy series from patients with AATD and the PiZZ genotype found liver disease to be present in the majority of patients, albeit unrecognized in many cases during lif e.

Source: Journal of Hepatology - March 20, 2018 Category: Gastroenterology Authors: Alice M. Turner, Jan Stolk, Robert Bals, Jason D. Lickliter, James Hamilton, Dawn R. Christianson, Bruce D. Given, Jonathan G. Burdon, Rohit Loomba, James K. Stoller, Jeffery H. Teckman Tags: Research Article Source Type: research