Treatment of α-1 antitrypsin deficiency using hepatic-specified cells derived from human-induced pluripotent stem cells.

ABSTRACT:

Objective

α-1 antitrypsin deficiency (AATD) is an inherited liver disease characterized by the "Z" mutations, which can cause pulmonary emphysema and liver fibrosis. Transplantation of the organ (i.e., the lung/liver) is the best treatment method, however, the scarcity of suitable donors limits its application. The cell transplantation technique poses an alternative way of combating liver failure.

Methods

Hepatic specific differentiation of the human induced pluripotent stem cells (iPSCs) was initiated with 100 ng/mL activin A, followed by 20 ng/mL of BMP-4 and 10 ng/mL of FGF-2. The cells were transplanted into the livers of AATD transgenic mice using intra-splenic injections. FK506 was used as an immunosuppressor. At 1, 3, and 6 months post-transplantation, the human serum

SUBMITTER: Chen Y 

PROVIDER: S-EPMC8129336 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature