Question

Tyrone has Gaucher disease. He receives recombinant glucocerebrosidase every other week in an infusion to prevent the symptoms. This treatment is an example of:

a) Gene therapy
b) Enzyme replacement therapy
c) Stem cell therapy
d) Immunotherapy

Answer 1

Tyrone's treatment with recombinant glucocerebrosidase for Gaucher disease is an example of enzyme replacement therapy, which supplies the enzyme that the patient's body cannot produce due to a genetic mutation.

Step-by-step explanation:

Tyrone receives recombinant glucocerebrosidase every other week in an infusion to prevent the symptoms of Gaucher disease. This treatment is an example of enzyme replacement therapy (ERT). ERT involves providing the patient with the enzyme that their body is deficient in or lacks due to a genetic mutation. This is different from gene therapy, where a non-mutated gene is introduced into the patient's cells to address a defective gene, often using a viral vector. In the provided examples, gene therapy is discussed in the context of treating conditions like ADA deficiency by inserting a functional gene into a patient's cells. However, in Tyrone's case, the treatment involves the direct supply of the vital enzyme itself, not the patient's genetic material.