Up Close: Unlocking the Basic Mechanisms of Lysosomal Storage Diseases
Lysosomal enzymes catalyze the breakdown of macromolecules in the cell. In humans, loss of activity of a lysosomal enzyme leads to an inherited metabolic defect known as a lysosomal storage disorder. The image above shows the structure of the human GALNS protein. The active site on the protein is marked by a magenta arrow. The specific shape of the active site allows the enzyme to select specific macromolecules in the cell for degradation. Structural biologist Scott Garman and his colleagues (Biochemistry and Molecular Biology) are unlocking the structure of these molecules, which is key to developing treatments for these diseases. Learn more.
Photo credit: Scott C. Garman