gene therapy and stem cells treatment for genetic cirrhosis

A recent paper from Nature journal reveals a big success in combining gene therapy with stem cells technologies in the treatment of a genetic type of cirrhosis. Researchers from  the Wellcome Trust Sanger Institute and the University of Cambridge have corrected a point mutation in Alpha1-antitrypsin gene with a "molecular scalpel". This mutation prevented anitrypsin from doing its job. Antitrypsin is an important inhibitor of trypsin and protects cells from enzymatic distruction.