Gene Therapy

Question

Somatic gene therapy aims to:

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1. **Objective**: Gene therapy is an experimental technique that uses genes to treat or prevent disease.
2. **Target Cells**: **Somatic gene therapy** involves inserting functional genes into the **somatic (body) cells** of a patient ($e.g.,$ bone marrow cells, lung cells).
3. **Effect**: It corrects the genetic defect only in that individual and the changes are **not passed on** to offspring.
4. **Example**: Treatment of ADA deficiency by inserting a functional $ADA$ gene into the patient's lymphocytes.
5. **Germ-line therapy**: This would involve changes in sperm or egg cells, which would be heritable (currently restricted in humans).

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Gene Therapy

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