It has been almost twenty years since scientists discovered the gene mutation responsible for cystic fibrosis. In this time, the life expectancy of those affected has improved from ten years of age to fifty. This can be attributed to a greater understanding of the genetics behind the disease, early detection and increased quality of treatment. Most current treatments for cystic fibrosis involve a mixture of aggressive use of antibiotics and regular physiotherapy. However, this is not a cure, it is just treatment of the disease.
A promising development has arisen in the area of gene therapy, with researchers currently working towards a cure, derived from the manipulation of DNA.
New gene therapies aim towards replacing the defective cells in the lungs with normal, healthy cells, through a method of replacing the DNA contained in these cells. Researchers are hopeful that this will be achieved through a spray which, when inhaled, can transfer normal DNA into the cells via microscopic vectors. The problem being encountered currently is the vector used to transfer genetic information. A viral vector, while effective, invokes an immune response, which in cystic fibrosis patients can be extremely dangerous. A non-viral vector, while much safer, is very inefficient.
Current research is promising, and it seems that a cure for the debilitating disease of cystic fibrosis is not too far in the future.
Article: Yoong T. & Jaffe A., "Cystic Fibrosis- An Update", Paediatrics & Child Health, Vol. 4 January 2009, pg 28 - 30.
