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Revision as of 23:01, 9 September 2018
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