Which conditions are known to be treated by gene therapy?

• A. Diabetes and hypertension
• B. Asthma and allergies
• C. Hemophilia and severe combined immunodeficiency
• D. Obesity and cardiovascular disease

Answer: (C)

Gene therapy is an innovative approach to treating genetic disorders by repairing, replacing, or supplementing faulty genes with functional ones. Conditions that can be effectively addressed by this method are primarily congenital or genetic diseases where specific genes are responsible for the disorder.

Hemophilia is a genetic disorder where blood does not clot properly due to the lack of certain clotting factors, which are proteins encoded by specific genes. Gene therapy has shown promise in treating hemophilia by delivering normal copies of the gene responsible for producing the deficient clotting factor, thereby restoring the ability to produce this protein.

Severe combined immunodeficiency (SCID), often known as "bubble boy disease," is another condition treatable by gene therapy. SCID results from mutations affecting the immune system, leaving individuals highly susceptible to infections. Gene therapy can be utilized to introduce a functional copy of the mutated gene, thereby enabling the production of healthy immune cells.

In contrast, conditions like diabetes, hypertension, asthma, allergies, obesity, and cardiovascular disease are typically multifactorial and influenced by a combination of genetic and environmental factors. Current gene therapy techniques are not yet applicable to these diseases as they involve complex interactions rather than a direct genetic defect. Therefore, gene therapy is currently focused on explicit genetic disorders