Gene Therapy: History and Future

A therapy used to treat the genetic disorders in human beings is termed as gene therapy. Gene therapy has the potential to bring about tremendous changes in treatment of various diseases and disorders. The therapy relies on gene replacement as the solution to treat the disorders. The genes that are responsible for the disease are removed or replaced with a 'healthy' or working gene. Thus, the enzyme or protein requirement of the body is fulfilled. It results into the elimination of the disease.

Gene Therapy History
The double-helix model of DNA, was proposed by Watson and Creek in the year 1953. The concepts of chromosomes, genes, etc. developed thereafter. It opened a whole new vista of possibilities and opportunities in the treatment of various hereditary diseases. The research in the field of human genetics acquired speed during the 1980s.

The first person to be treated by gene therapy was a four year old girl (name not known) from the United States. The lack of production, of adenosine deaminase (ADA), had made her immune system weak. Thus, she had become susceptible to many severe diseases. The girl was treated on 14th September, 1990, at the National Institutes of Health's Clinical Center, Bathesda, Maryland. Dr. W. French Anderson and his colleagues at the health center, carried out the proceedings. White blood cells were extracted from the body. After the implantation of genes that produce ADA, the cells were transferred back to the girl's body. Considerable improvement in the immune system of the girl was noticed.

Meanwhile, the trials of gene therapy continued on various diseases. The patients with skin cancer, melanoma were treated by means of gene therapy. Attempts were made to treat cystic fibrosis with the gene therapy. Cystic fibrosis is a disease which affects the airways present in the respiratory system. However, the process of using gene therapy was complicated in this case.

Gene Therapy Future
The future of gene therapy holds promises in the treatment of many incurable diseases. The problem with gene therapy is that, the treatment sounds promising theoretically, however, is difficult to implement. Gene therapy involves a whole lot of complicated set of activities involving tissue targeting, cellular trafficking, delivery of genes to organs, safety of the vector, activity of therapeutic protein, etc. The above mentioned activities being understood incompletely, the progress in the field of gene therapy seems to be slow.

For gene therapy to be implemented, certain prerequisites or basic things are mandatory. Therapeutically suitable genes should be available for the treatment. For the delivery of the genes, vectors are needed. These may be viral or non-viral vectors. For the therapy to be successful, the efficacy and safety of the method should be determined or established in advance.

The diseases to be treated with gene therapy in the future will be the cardiovascular diseases, monogenic diseases and the haemophilias. Despite the current advancements and future research in the field of gene therapy, it should be understood that there won't be a standard cure for diseases. The different nature of different diseases makes it necessary to overcome new technical and medical hurdles for every new disease.

The pace at which technology is improving and the knowledge about the body systems is being accumulated, the future of gene therapy looks bright.