Nanotechnology applied to treatment of mucopolysaccharidoses

摘要

Mucopolysaccharidoses (MPS) are a group of rare genetic lysosomal storage diseases caused by deficiencies of enzymes involved in the catabolism of glycosaminoglycans (GAGs). The lack of enzymes causes GAG accumulation in the lysosomes, resulting in generalized cell, tissue, and organ dysfunctions.