Human iPS Cell-based Liver-like Tissue Engineering at Extrahepatic Sites in Mice as a New Cell Therapy for Hemophilia B.

In this study, we attempted to show the effectiveness of human iPS cell-based liver-like tissue engineering at an extrahepatic site for treatment of hemophilia B, also called factor IX (FIX) deficiency. HLCs were transplanted under the kidney capsule where the transplanted cells could be efficiently engrafted. Ten weeks after the transplantation, human albumin (253 μg/mL) and α-1 antitrypsin (1.2 μg/mL) could be detected in the serum of transplanted mice. HLCs were transplanted under the kidney capsule of FIX-deficient mice. The clotting activities in the transplanted mice were approximately 5% of those in wild-type mice. The bleeding time in transplanted mice was shorter than that in the nontransplanted mice. Taken together, these results indicate the success in generating functional liver-like tissues under the kidney capsule by using human iPS cell-derived HLCs. We also demonstrated that the human iPS cell-based liver-like tissue engineering technology would be an effective treatment of genetic liver disease including hemophilia B.
PMID: 29637813 [PubMed - in process]

Source: Cell Transplantation - February 1, 2018 Category: Cytology Authors: Okamoto R, Takayama K, Akita N, Nagamoto Y, Hosokawa D, Iizuka S, Sakurai F, Suemizu H, Ohashi K, Mizuguchi H Tags: Cell Transplant Source Type: research