Question

Enlist the uses of gene therapy.

Hint:

The core principle of gene therapy is simple: "replace a bad gene with a good gene." It aims to fix the root cause of a disease at the DNA level rather than just treating the symptoms.

Answer 1

Gene Therapy

Gene therapy is a modern medical technique used to treat or prevent diseases by correcting the underlying genetic problem. It works by introducing, altering, or repairing genetic material inside a patient’s cells.

Uses of Gene Therapy:

• Treating single-gene disorders: Applied in inherited diseases caused by a single faulty gene, such as:
  • Severe Combined Immunodeficiency (SCID)
  • Cystic Fibrosis
  • Hemophilia
  • Sickle Cell Anemia
• Cancer treatment: Used to modify immune cells (e.g., T-cells in CAR-T therapy) to recognize and destroy cancer cells. It can also introduce suicide genes into cancer cells, making them self-destruct.
• Treating viral infections: Ongoing research explores ways to make cells resistant to viruses like HIV by modifying their genetic makeup.
• Treating degenerative diseases: Promising applications include replacing or repairing defective genes in diseases such as:
  • Parkinson’s disease
  • Huntington’s disease
  • Muscular dystrophy
• Vaccine development: DNA vaccines, a form of gene therapy, can trigger a strong and long-lasting immune response.