Answer: Gene therapy means correcting a genetic defect by introducing a normal (functional) gene into the cells of a patient. Its uses:
- Treatment of inherited single-gene disorders such as SCID (adenosine deaminase deficiency), cystic fibrosis, haemophilia, thalassaemia and sickle-cell anaemia.
- Replacing a defective or missing gene with a functional gene so that the missing protein/enzyme is produced.
- Treatment of some cancers (e.g. making tumour cells sensitive to drugs or boosting immune cells against them).
- Possible treatment of some viral diseases (e.g. AIDS) and degenerative disorders.
- Germ-line gene therapy can in principle prevent the transfer of a defective gene to the next generation.
