Division of Microbiology, Department of Pathology and Microbiology, Nihon University School of Medicine, Tokyo, Japan.
Department of Pediatrics and Child Health, Nihon University School of Medicine, Tokyo, Japan.
Stem Cells Transl Med. 2021 Jun;10(6):829-835. doi: 10.1002/sctm.20-0225. Epub 2021 Feb 6.
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.
先天性代谢疾病是一组由单一特定酶活性缺乏引起的遗传性疾病。如果没有适当的治疗，受影响的患者会遭受严重的神经功能障碍和最终死亡。目前的治疗方法主要是试图减缓疾病的进展，但不能治愈疾病。这些疾病中有几种已经证明从肝移植中获益匪浅；然而，这种方法受到与该侵袭性手术相关的发病率和供体器官短缺的限制。因此，有必要为这些患者建立提高生活质量的新策略。一种潜在的解决方案是使用干细胞生成的肝细胞进行再生治疗。在此，我们讨论了胎盘干细胞之一的人羊膜上皮细胞在临床应用中所必需的相关问题。以枫糖尿症为例，肝脏替代是一种有效的治疗方法，我们从临床医生的角度探讨了这种方法。
