Gene Therapy

1. General Features

Gene therapy is a highly experimental technique that has shown promise in some clinical trials in humans for diseases such as immunodeficiencies (where the immune system does not function properly), muscular dystrophy (progressive loss of skeletal muscle) and anaemia (decreases in the number or function of red blood cells).

The gene therapy approach is useful for diseases in which a single gene is affected, either by a single mutation or multiple mutations in the same gene. In diseases which involve multiple genes, this approach is much more complicated and is unlikely to work.