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What is Gene Therapy?

Gene therapy is a medical technique that uses genes to treat or prevent diseases. It works by either replacing a faulty gene or adding a healthy version of a gene to a person’s cells. This treatment is used to address genetic disorders like sickle cell disease, cystic fibrosis, and certain types of cancer by targeting the underlying genetic cause. Gene therapy can be delivered in various ways, including by injecting genes directly into the patient's body or by modifying cells outside the body and then reintroducing them.


What Diseases are Cured by Gene Therapy?


Gene therapy has shown potential in treating and curing a variety of diseases, particularly those caused by genetic mutations. Some of the conditions that gene therapy can cure or effectively treat include:

  • Severe Combined Immunodeficiency (SCID): A genetic disorder causing a weakened immune system.

  • Hemophilia and Blood Disorders: Conditions where blood clotting is impaired due to missing or defective genes.

  • Blindness from Retinitis Pigmentosa: A degenerative eye disease leading to vision loss.

  • Leukemia: A type of cancer affecting the blood and bone marrow.

  • Inherited Neurological Disorders: Such as spinal muscular atrophy (SMA) and Huntington’s disease.

  • Cancer: Gene therapy can be used to target and treat specific cancer cells.

  • Heart and Blood Vessel Diseases: Gene therapy may aid in treating conditions like coronary artery disease by promoting angiogenesis.