How Lysosomes Keep Us Healthy

Abstract

Lysosomes are specialized cell structures that serve as the garbage collection system of the body‘s cells. The lysosome contains components that clean up various types of cellular “trash” and are responsible for activating the recycling process. When lysosomes do not work properly, trash accumulates in the cells, eventually leading to a sick cell and cell death. This can lead to the development of several lysosomal storage diseases. Symptoms of these diseases vary in severity, from symptoms that barely affect a patient’s life up to symptoms already starting at birth that greatly reduce life expectancy. Two treatment options are available for some of these diseases that can ameliorate symptoms, the enzyme replacement therapy and substrate reduction therapy. A new research approach called gene therapy, provides a potential cure. In this article, we will explain the role of lysosomes and what happens when they do not work properly. We will also provide details about the available treatments and how gene therapy could be a breakthrough in the field.