Q.Gene therapy is an attempt to correct a Genetic defect by providing a normal gene into the individual. By this the function can be restored. An alternate method would be to provide gene product known as enzyme replacement therapy, which would also restore the function. Which in your opinion is a better option? Give reasons for your answer.

Step 1. Compare what each approach actually does: enzyme replacement therapy supplies the missing gene PRODUCT (the functional enzyme) directly to the patient, restoring function only for as long as that dose of enzyme remains active in the body, whereas gene therapy supplies the missing GENE itself, so the patient's own (engineered) cells can go on producing the protein internally. Step 2. Note the practical consequence: because injected enzyme is eventually broken down and cleared, enzyme replacement therapy demands repeated, essentially lifelong dosing, with the associated cost and treatment burden that implies; gene therapy, once the corrected gene is properly integrated and expressed, allows the corrected cells to keep producing the enzyme on their own, at least for as long as those cells survive. Step 3. Acknowledge the current limitation of gene therapy honestly: in practice, standard somatic-cell gene therapy (as used for ADA-SCID) still needs periodic infusions too, because the engineered lymphocytes are not immortal — so today's gene therapy is not automatically a single, permanent fix either. Step 4. Give the reasoned conclusion: gene therapy is still the better option in principle, because it has a genuine path to a PERMANENT cure (the chapter not …

Compare the mechanisms (correcting the gene vs. replacing its product) and their consequences for permanence, using the chapter's own honest …

• Claiming gene therapy is already a guaranteed one-time permanent cure, ignoring the chapter's own point that standard somatic-cell gene therapy still needs periodic infusions. …