Matas A J, Sutherland D E, Steffes M W, Mauer S M, Sowe A, Simmons R L, Najarian J S
Science. 1976 May 28;192(4242):892-4. doi: 10.1126/science.818706.
A sustained decrease of plasma bilirubin concentrations occurred in homozygous recessive Gunn rats lacking the enzyme uridine diphosphate glucuronyltransferase following infusion into the portal vein of hepatocytes from heterozygous nonjaundiced Gunn rats possessing the enzyme. Transplantation of cells capable of continuous enzyme production could be an effective mode of therapy for congenital enzyme deficiency diseases.
在缺乏尿苷二磷酸葡萄糖醛酸转移酶的纯合隐性Gunn大鼠中,门静脉输注来自具有该酶的杂合非黄疸Gunn大鼠的肝细胞后,血浆胆红素浓度持续下降。移植能够持续产生酶的细胞可能是治疗先天性酶缺乏疾病的一种有效治疗方式。