Enlist any four uses of gene therapy.

Answer: Gene therapy is the correction of a genetic defect by introducing a normal (functional) gene into the cells of a patient. Uses:

1. Treatment of SCID (severe combined immunodeficiency) caused by ADA (adenosine deaminase) deficiency; the first gene therapy (1990) was for ADA deficiency.
2. Treatment of single-gene disorders such as cystic fibrosis, haemophilia and thalassaemia / sickle-cell anaemia.
3. Treatment of certain cancers, e.g. by inserting genes that make tumour cells sensitive to drugs or that strengthen immune cells against the tumour.
4. Treatment of some acquired diseases such as AIDS and of degenerative disorders such as Parkinson’s disease and muscular dystrophy.