戈谢病中的骨髓移植：混合嵌合状态的影响。
Bone marrow transplantation in Gaucher's disease: effect of mixed chimeric state.
作者信息
Chan K W, Wong L T, Applegarth D, Davidson A G
机构信息
Department of Paediatrics, B.C.'s Children's Hospital, Vancouver, Canada.
出版信息
Bone Marrow Transplant. 1994 Aug;14(2):327-30.
PMID:7994251
Abstract
The effective dose and schedule of enzyme replacement therapy for Gaucher's disease have not been definitely established. We report a case of mixed chimeric state in an allogeneic BMT patient and followed her clinical and laboratory progress. The result shows that a low but sustained glucocerebrosidase level may provide symptomatic relief for this lysosomal disorder.
摘要
戈谢病酶替代疗法的有效剂量和疗程尚未明确确定。我们报告了1例异基因骨髓移植患者出现混合嵌合状态的病例，并对其临床和实验室进展进行了随访。结果显示，低水平但持续的葡萄糖脑苷脂酶可能为这种溶酶体疾病提供症状缓解。
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