Chronic Granulomatous Disease is a diverse group of hereditary diseases in which there is a mutation in a single gene that encodes for the production of sub-units of the enzyme NADPH oxidase. This molecule stimulates the body's ability to fight off invading micro-organisms such as bacteria, by turning oxygen molecules (O2) …show more content…
Therapeutic DNA needs to be functional in the long-term and the cells containing it need to be long-lived and stable if the therapy is going to provide a permanent cure. However, difficulty integrating the DNA into the genome combined with the fact that many cells divide so rapidly means long-term benefits are problematic to achieve and repeated treatments are needed.
Disorders arising from one gene mutation are the most promising candidates for gene therapy but a lot of the most common disorders such as diabetes, heart disease and arthritis are caused by a combination of altered genes making them particularly difficult to treat.
The body's immune system can respond to the modified vectors and disrupt the effectiveness of gene therapy. The immune system's recognition of foreign bodies also means repeated therapy can become problematic.
Use of the viral vectors can also pose a risk to patients in a variety of ways by triggering toxic, immune or inflammatory reactions or through the virus itself recovering its ability to cause disease once inside the