What are Some Applications of Enzyme Engineering in Neonatal Disorders?
One notable application is in the development of enzyme replacement therapies (ERTs). For instance, Pompe disease is a rare genetic disorder caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). Engineered versions of GAA can be administered to patients to improve glycogen breakdown, thereby mitigating the disease's effects. Similarly, enzyme engineering can help in developing gene therapy approaches, where genes encoding the engineered enzymes are delivered to the patient's cells to restore normal function.
