Gene therapy is given in aEUR’
**Core Concept**
Gene therapy is a medical intervention that aims to treat or prevent diseases by modifying a person's genes. It involves the introduction of healthy copies of a gene into a patient's cells to replace faulty or missing genes. This approach has shown promise in treating genetic disorders, cancer, and infectious diseases.
**Why the Correct Answer is Right**
The correct answer is **C. Severe Combined Immunodeficiency (SCID)**. Gene therapy is used to treat SCID, a rare genetic disorder characterized by a severe deficiency in the immune system. In SCID, the adenosine deaminase (ADA) gene is mutated, leading to the accumulation of toxic substances that destroy immune cells. By introducing a healthy copy of the ADA gene into a patient's stem cells, gene therapy can restore immune function and improve the patient's quality of life.
**Why Each Wrong Option is Incorrect**
**Option A:** Sickle cell anemia is typically treated with pharmacological interventions, such as hydroxyurea, and blood transfusions, rather than gene therapy.
**Option B:** Cystic fibrosis is being explored as a potential target for gene therapy, but it is not the most common indication for this treatment.
**Option D:** Cancer gene therapy is being researched, but it is not typically used to treat cancer in the same way as SCID.
**Clinical Pearl / High-Yield Fact**
Gene therapy has shown promise in treating genetic disorders, but it is not without risks, and long-term safety and efficacy data are still being collected.
**Correct Answer: C. Severe Combined Immunodeficiency (SCID).