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 could 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.
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Start your 14-day free trial to unlock the full solution →Gene therapy is generally favoured over enzyme replacement therapy because it addresses the root genetic cause of the disorder rather than merely and repeatedly treating its symptoms.
In gene therapy, a normal, functional gene is introduced into an individual's cells (using a vector, commonly a modified virus) to compensate for or correct the effect of a defective/non-functional gene, aiming to restore the missing gene product's function at its biological source. In enzyme replacement therapy, the missing or defective enzyme (the gene's product) is itself supplied directly to the patient, typically by repeated injection, to compensate for the deficiency. While enzyme replacement therapy can be effective and is sometimes preferable when gene delivery is technically difficult or risky, gene therapy is generally considered the more fundamentally advantageous option because it targets the actual genetic defect rather than only its downstream consequence: a successful gene-therapy intervention (especially one that modifies stem cells, whose descendants continue producing the correct gene product) could, in principle, provide a long-term or even permanent cure from a single treatment, whereas enzyme replacement provides only temporary relief and must be administered repeatedly (often for the patient's entire life), can be costly on an ongoing basis, …
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