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

Gene therapy aims to fix the root cause by inserting a functional gene into the patient's cells, so those cells themselves produce the missing protein continuously. Once the gene integrates and expresses, the correction is long-lasting—potentially permanent if germ-line or stable somatic integration occurs—and the body's own machinery takes over. The patient no longer depends on repeated external doses.

Enzyme replacement therapy, on the other hand, supplies the protein directly from outside. It works immediately and avoids the risks of gene insertion (immune rejection, insertional mutagenesis, or unpredictable expression), but the effect is temporary. The enzyme degrades, so the patient needs regular, often lifelong infusions. It treats the symptom, not the underlying genetic defect. …

The question asks us to weigh two fundamentally different approaches to treating genetic disorders. One targets the cause, the other manages the symptom. Understanding this distinction is the key to forming a reasoned opinion.

Gene therapy works at the DNA level. When a person inherits a defective gene that fails to produce a functional protein, gene therapy introduces a correct copy of that gene into the patient's cells. Once integrated, those cells can manufacture the protein themselves. The appeal is obvious: if successful, you've corrected the underlying defect. The patient's own cells become the factory for the missing protein, potentially for life. This is especially attractive for single-gene disorders where one faulty gene is responsible for the entire disease.

Enzyme replacement therapy, by contrast, is a workaround. You supply the missing enzyme or protein directly, usually through regular injections or infusions. The body uses what you've given it, but the effect is temporary. The protein degrades, gets used up, and you need another dose. It's management, not cure. The genetic defect remains untouched, passed on to the next generation if the patient has children.

So which is better? In principle, gene therapy holds the greater promise. Here's why:

• Permanence: A single successful gene therapy treatment could last a lifetime, whereas enzyme replacement requires lifelong, repeated administration. The patient is freed from a perpetual treatment schedule.
• Cost over time: Though gene therapy may be expensive upfront, enzyme replacement accumulates costs year after year. Over decades, the financial burden can be staggering.
• Quality of life: Continuous infusions or injections are invasive and time-consuming. Gene therapy, if it works, eliminates this burden entirely.
• Physiological advantage: The corrected gene produces protein exactly when and where the body needs it, in response to normal regulatory signals. Externally supplied enzyme may not reach all tissues effectively or may be present in unnatural concentrations.

But we must acknowledge the practical reality. Gene therapy, despite its elegance, faces significant hurdles. Delivering the gene to the right cells, ensuring stable integration without disrupting other genes, avoiding immune rejection of the vector — these are not trivial problems. Many gene therapy trials are still experimental. Enzyme replacement therapy, though imperfect, is a proven, relatively safe option available now for several disorders like Gaucher disease or Fabry disease. …

Method — compare on 3 axes: cause, permanence, cost of repeat