SERPINA1 and sickle cell disease: ZNF system showed promising results in gene therapy of the mutation causing sickle cell anemia in human iPSCs.16 Further, the bi-allelic correction of the point mutation (Glu342Lys) in the α1-antitrypsin gene (A1AT or SERPINA1) by ZNF system and subsequent re-introduction of differentiated iPSCs into the liver of the recipient mouse resulted in the restoration of structure and function of A1AT both in vitro and in vivo.