Clinical perspective on the use of human amniotic epithelial cells to treat congenital metabolic diseases with a focus on maple syrup urine disease

Stem Cells Translational Medicine

Brendan H. Grubbs¹  Mika Ishige²  Ichiro Morioka²  Erika Ogawa²  Toshio Miki³  Satoshi Hayakawa⁴  Chika Takano⁴ 

[1]Department of Obstetrics and Gynecology Keck School of Medicine, University of Southern California Los Angeles California USA

[2]Department of Pediatrics and Child Health Nihon University School of Medicine Tokyo Japan

[3]Department of Physiology Nihon University School of Medicine Tokyo Japan

[4]Division of Microbiology, Department of Pathology and Microbiology Nihon University School of Medicine Tokyo Japan

关键词: cell transplantation;    cellular therapy;    liver regeneration;    placenta;    stem cell transplantation;   

DOI  :  10.1002/sctm.20-0225

来源: DOAJ

【 摘 要 】

Abstract Congenital metabolic diseases are a group of hereditary disorders caused by the deficiency of a single specific enzyme activity. Without appropriate therapy, affected patients suffer severe neurologic disability and eventual death. The current mainstays of management attempt to slow disease progression, but are not curative. Several of these diseases have demonstrated significant benefits from liver transplantation; however, this approach is limited by the morbidity associated with this invasive procedure and a shortage of donor organs. Therefore, there is a need to establish a new strategy for improving the quality of a life for these patients. One potential solution is regenerative therapy using hepatocytes generated from stem cells. Herein, we discuss pertinent issues necessary for clinical application of the human amniotic epithelial cell, a type of placental stem cell. Focusing on maple syrup urine disease as an example, where liver replacement is an effective therapy, we explore this approach from a clinician's perspective.

【 授权许可】

Unknown