How Can Catalysis Help in Treating Genetic Disorders?
Understanding the role of catalysis in genetic disorders opens the door to various treatment strategies. Enzyme replacement therapy (ERT) is one approach, where the defective enzyme is replaced with a functional one. For instance, in Gaucher's disease, patients can receive intravenous infusions of recombinant glucocerebrosidase to break down accumulated substrates. Another approach is the use of enzyme activators or chaperone molecules that enhance the function of the residual enzyme activity in patients. These small molecules stabilize the enzyme, improving its catalytic activity and reducing the accumulation of toxic substrates.
