The lysosome: a role in disease.

Abstract

Increasing interest in elucidating the role of the lysosome in intracellular metabolic economy has resulted in a deeper understanding of some inborn errors of metabolism, as well as a clearer definition of the intracellular vacuolar apparatus and the role of lysosomes in intracellular drug action and possible specific chemotherapeutic actions. The lysosomal system plays a key role in Pompe's disease (glycogenosis type II). The effect of a selective deficiency of lysosomal acid maltase (acid α-glucosidase) on muscle morphology and biochemistry is shown. The activity of other lysosomal acid hydrolases in muscle is significantly increased. Change in intracellular activity and translocation of primary and secondary lysosomes after membrane activation by steroids are discussed, with data showing the intranuclear migration of lysosomal-like particles, suggesting a mechanism for template activation. Lysosomes play a decisive role in initiating nucleocytoplasmic exchange of information, and they may play a role in genetic derepression, with obvious repercussions in normal and abnormal cell growth. The lysosome model in reference to acid hydrolase induction, especially endocytic and phagolysosomal activity, has been examined.