Team:ASIJ Tokyo

A1-AT
deLIVERy

Using Stem Cell and CRISPR Technology to Combat
Alpha-1 Antitrypsin Deficiency

ABSTRACT

Alpha-1 Antitrypsin deficiency is a common genetic disorder -- the defective gene for which is carried by 1 in 25 people -- which arises from a single base pair mutation in the SERPINA1 gene, resulting in the production of a form of antitrypsin prone to polymerization. The mutated antitrypsin then builds up in liver cells and is unable to inhibit proteases in the lung