Rennert O M
Ann Clin Lab Sci. 1975 Sep-Oct;5(5):355-62.
The genetic mucopolysaccharidoses represent the group of disorders recognized in the early 1900's. In half a century, it was recognized that these were disorders of polyanionic macromolecules known as glycosaminoglycans. Within the past five years, these disorders have been identified as prototypes of lysosomal diseases, are a genetically heterogeneous group and this heterogeneity is recognizable in in vitro systems. Finally, these disorders represent prototypes for the development of methods of enzyme replacement therapy.
遗传性黏多糖贮积症是20世纪初被确认的一组病症。在半个世纪的时间里，人们认识到这些是被称为糖胺聚糖的聚阴离子大分子的病症。在过去五年中，这些病症已被确定为溶酶体疾病的原型，是一组基因异质性疾病，并且这种异质性在体外系统中是可识别的。最后，这些病症代表了酶替代疗法方法开发的原型。
