Gene therapy can correct defective genes, treat or prevent genetic disorders, and potentially cure conditions like cystic fibrosis, sickle cell anemia, and some forms of cancer. It offers a targeted approach with long-lasting effects.
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Gene therapy offers potential benefits in treating genetic disorders by addressing the root cause of the condition rather than just managing symptoms. It involves introducing, removing, or altering genetic material within a patient's cells to correct faulty genes or compensate for genetic deficiencies. This approach could provide long-term or permanent cures for disorders such as cystic fibrosis, sickle cell anemia, and certain inherited forms of blindness, potentially reducing the need for ongoing treatments or interventions and improving quality of life.