Howell J M, Dorling P R, Shelton J N, Taylor E G, Palmer D G, Di Marco P N
School of Veterinary Studies, Murdoch University, Perth, Western Australia.
Neuromuscul Disord. 1991;1(6):449-54. doi: 10.1016/0960-8966(91)90008-g.
Adding acid alpha-glucosidase to cultures of Pompe's disease muscle has resulted in enzyme uptake and reduction in concentration of glycogen. However, bone marrow transplantation has been unsuccessful as a treatment. Immune rejection may have contributed to this failure. Twin calves share a placenta and carry lymphoreticular cells of each other's type, they become lymphoreticular chimeras in utero and immune rejection does not occur. One natural and three sets of twins produced by embryo transfer were studied in Pompe's disease cattle. Chimerism persisted throughout life and the situation was analogous to a transplant of histocompatible bone marrow stem cells. The activity of acid alpha-glucosidase in leucocytes and in biopsies of the semitendinosus muscle and the mean activity in diaphragm, spleen and lymph node obtained after death from affected twins were significantly higher than in single affected calves. Glycogen concentration was lowered in liver, spleen and lymph node but not in muscles. The affected twins showed clinical signs and changes in muscle similar to those seen in affected single calves. It is concluded that bone marrow transplantation is unlikely to be a successful treatment for Pompe's disease.
向庞贝氏病肌肉培养物中添加酸性α-葡萄糖苷酶已导致酶摄取并降低了糖原浓度。然而，骨髓移植作为一种治疗方法并不成功。免疫排斥可能是导致这种失败的原因。双胎小牛共享一个胎盘，并携带彼此类型的淋巴网状细胞，它们在子宫内成为淋巴网状嵌合体，不会发生免疫排斥。对庞贝氏病牛中自然产生的一对双胞胎和通过胚胎移植产生的三组双胞胎进行了研究。嵌合现象持续终生，这种情况类似于组织相容性骨髓干细胞的移植。受影响双胞胎死后获得的白细胞、半腱肌活检组织中酸性α-葡萄糖苷酶的活性以及膈肌、脾脏和淋巴结中的平均活性明显高于单只患病小牛。肝脏、脾脏和淋巴结中的糖原浓度降低，但肌肉中未降低。受影响的双胞胎表现出与受影响的单只小牛相似的临床症状和肌肉变化。得出的结论是，骨髓移植不太可能成为庞贝氏病的成功治疗方法。
