Syndrome of the defective lysosome--the genetic mucopolysaccharidoses

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.