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

Author:

Takano Chika12,Grubbs Brendan H.3,Ishige Mika2,Ogawa Erika2,Morioka Ichiro2,Hayakawa Satoshi1,Miki Toshio4

Affiliation:

1. Division of Microbiology, Department of Pathology and Microbiology Nihon University School of Medicine, Tokyo, Japan

2. Department of Pediatrics and Child Health Nihon University School of Medicine, Tokyo, Japan

3. Department of Obstetrics and Gynecology Keck School of Medicine, University of Southern California, Los Angeles, California, USA

4. Department of Physiology Nihon University School of Medicine, Tokyo, Japan

Abstract

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.

Funder

Overseas Scholarship of Graduated Student of Nihon University School of Medicine

Publisher

Oxford University Press (OUP)

Subject

Cell Biology,Developmental Biology,General Medicine

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