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 a lot of changes in the treatment of quite a few diseases and disorders. The therapy relies on gene replacement as the solution to treat the disorders.

The genes responsible for causing 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. Let us find more on gene therapy's history and future.

• 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 for the treatment of various hereditary diseases. Research in the field of human genetics gained speed in the 1980s.

• The first person to be treated with gene therapy was a four-year-old girl (name not known) from the United States. The lack of production of adenosine deaminase (ADA) made her immune system weak. Therefore, she became susceptible to many severe diseases. 

• The girl was treated on 14th September, 1990 at the National Institutes of Health's Clinical Center, Bethesda, Maryland. Dr. W. French Anderson and his colleagues at the health center carried on with the proceedings during which the 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 was observed in the immune system of the girl.
• The trials of gene therapy continued for many different diseases. The patients suffering from a particular skin cancer called melanoma were treated with the help of gene therapy. 

Attempts were made for treating cystic fibrosis with gene therapy. Cystic fibrosis is a disease which affects the airways of the respiratory system. However, in this case the process used for implementing the gene therapy was quite complicated.

• The future of gene therapy holds promise in the treatment of many incurable diseases. At least, the focus for the time being is only on the incurable diseases. The problem with gene therapy is that the treatment sounds theoretically promising; however, it is quite difficult to implement.

• Also, the unavailability of approved products in the markets for a long time has deterred pharmaceutical companies from entering this field.
• Gene therapy involves a lot of complicated activities which involve tissue targeting, cellular trafficking, delivery of genes to organs, safety of vector, activity of therapeutic protein, etc. 

• Due to incomplete understanding of the above mentioned activities, progress in the field of gene therapy has been a bit slow. However, if all these problems are resolved then the development should take place at a faster pace.

To implement gene therapy, it is necessary that certain prerequisites are followed. Therapeutically suitable genes are needed for carrying out the treatment. Vectors are needed for the delivery of genes. The vectors could be viral or non-viral. For this to be successful, efficacy and safety of this method should be determined or established in advance.

• One of the challenges which lies before the development of gene therapy is the problems associated with transferring the right genetic material to the intended cells.
• The diseases to be treated with gene therapy in the future would mostly be the cardiovascular diseases, monogenic diseases and hemophilia.

• One should understand that despite the current advancements and future research in the field of gene therapy, there won't be a standard cure for the diseases. Also, the different nature of different diseases makes it important that we overcome the technical and medical hurdles that would arise for every new disease.

• According to Dr. David Russell from the University of Washington, the focus of gene therapy would shift from treatment of rare diseases to common health problems. Dr. Carl June from the University of Pennsylvania opines that it won't take more than 5 years for gene therapy to become a widespread treatment measure for a variety of diseases.

• It is speculated that gene therapy would result into the creation of a superior race. However, the underlying idea of using gene therapy is to provide a cure for the hereditary diseases. There are few who fear that using gene therapy is like manipulating the DNA; therefore, using gene therapy is also perceived as sinful by many.

• In reality, gene therapy is just like any other therapy. The option of somatic cell therapy is available for those who want to avail the benefits of gene therapy and at the same time don't want the genetic changes to pass on to the next generation.

It is a gene therapy which has received approval from the European Commission. The Glybera therapy was developed with the intention of treating the problem of familial lipoprotein lipase deficiency (LPLD). When the genes which code for lipoprotein Lipase (LPL) get damaged, this protein doesn't function in a proper manner.

The breakdown of fats does not take place smoothly and it causes the swelling of pancreas. Fat levels in the body rise above the expected/normal levels. The problem is therefore, treated with the help of gene therapy.

The in utero gene therapy is seen as one of the promising fields in the development of gene therapy. Genetic disorders responsible for causing damage to the fetus before birth can be prevented or at least, slowed down with the help of this therapy. Stem cells of the fetus allow access to the vectors that are used in gene therapies. This is one of the advantages of using the in utero gene therapy.

Going by the pace at which technology is developing and the knowledge about the bodily systems is being accumulated, the future of gene therapy looks bright. However, one should keep in mind that gene therapy demands utmost care and the right vision for its proper implementation.