Q: What is gene therapy? Illustrate how gene therapy can be used to treat genetic disorders with the help of suitable examples. (GS Paper III – Science & Technology | 150 words)

 

Q: What is gene therapy? Illustrate how gene therapy can be used to treat genetic disorders with the help of suitable examples.

(GS Paper III – Science & Technology | 150 words)

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Introduction:

Gene therapy is a medical technique that involves modifying or manipulating genes within an individual’s cells to treat or prevent disease. It offers a potential cure rather than symptom management, especially for inherited genetic disorders.

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How It Works:

• Gene therapy may involve:

  • Replacing a faulty gene with a healthy one.

  • Inactivating a malfunctioning gene.

  • Introducing a new gene to help fight a disease.

• Techniques often use viral vectors or CRISPR-Cas9 for gene delivery or editing.

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Examples of Application:

1. Spinal Muscular Atrophy (SMA):

   • Zolgensma, a gene therapy, replaces the missing SMN1 gene to stop muscle deterioration in infants.

2. Beta Thalassemia & Sickle Cell Anaemia:

   • CRISPR-based therapies like Exa-cel correct the defective gene in blood stem cells.

3. Leber’s Congenital Amaurosis:

   • An inherited eye disorder treated by delivering a normal RPE65 gene using Luxturna.

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Conclusion:

Gene therapy holds transformative potential for genetic diseases, cancers, and rare disorders. While challenges remain in terms of safety, delivery, and cost, it is a major leap in personalized and regenerative medicine.