Gene Therapy for Lysosomal Disorders

The lysosomal storage disorders (LSDs) represent a wide group of clinically diverse types of disorders that include the mucopolysaccharidoses, sphingolipidoses, and defects in glycoprotein catabolism (Neufeld, 1991). All are characterized by the inability of cellular lysosomes to degrade complex substrates, which stems from a deficiency in one or more lysosomal enzymes. Accumulation of undegraded macromolecules in the lysosomes of cells leads to massive lysosomal distension, anatomical distortion of cells, and functional impairment of affected tissues.