In the 60 years since Watson and Crick’s landmark discovery of the structure of DNA, our understanding of how genes influence disease has increased exponentially. For some conditions, an exciting therapeutic prospect exists: gene therapy. Gene therapy attempts to repair faulty genes instead of simply treating symptoms.
For many conditions, the exact genetic mechanisms underlying them have now been elucidated. While a lot of diseases are the result of a complex interaction between multiple genes and environmental factors, others are the result of a single mutation, resulting in the failure to produce an essential functional protein. For such conditions, an exciting therapeutic prospect exists: gene therapy. In principle, the idea behind gene therapy is very simple. Whereas conventional medicine generally attempts to replace the missing gene product or repair the damage caused by its absence, gene therapy attempts to repair the faulty gene itself. Why treat symptoms when you can treat the cause?