An Introduction to Gene Therapy

The field of medicine has improved greatly with the recent advancement of science and technology. Diseases that were thought to be incurable are now cured with these new discoveries. Gene therapy and nanotechnology are two of the latest technologies that are constantly under development. Gene therapy is where scientists use several theoretical approaches to treat genetic disorders. Some diseases are treated by removing the defective gene from the genome. Sometimes the defective genes are inactivated. Introduction of new genes is also used in gene therapy.

How gene therapy differs from other methods of treatment

There are many genetic disorders among human beings. They occur as a result of defects in the genetic materials. Some diseases occur as a result of defective function of a certain gene, while some occur due to defective structure. Genetic disorders were thought to be incurable in the past because there was no way to alter the structure of genetic materials that are situated within cells. Some of these genetic disorders are now cured with the discovery of gene therapy.

Gene therapy is the technology of alteration of the structure and function of the genes. Genetic materials are contained within almost all the cells of the body. As a result, defective genetic materials are expressed in most of the cells. Therefore, in order to cure a genetic disease, there should be a way to approach all the cells. Scientists have discovered various methods to achieve this goal.

Vectors of gene delivery

Virus vectors are used as vehicles for delivering genes to the involved cells. Viruses can spread throughout the body. They are designed to enter the cells and incorporate their genetic materials into the cells’ genome. Then the viruses use the cells to replicate themselves. Scientists have used these properties of viruses to achieve their goals in gene therapy.

First they remove the harmful parts of the virus and replace them with the gene that they want to deliver to the cells. Thereafter, they introduce the virus to the human body. The virus then enters the required cell and introduces the new gene to the cell. This is a successful method of delivery of genes, but it also has its own drawbacks. Viruses are small particles. Sometimes the involved genes are very large. In such situations, viruses cannot carry the genes. Therefore, scientists started looking for other vectors for the delivery of genes to the cells. They are called non-viral vectors.

Non viral vectors are used to deliver larger genes into cells, including plasmids. Plasmids are circular DNA molecules. They are found in bacteria. Bacteria use these plasmids to exchange genetic materials between one another. They can overcome the obstacles faced by viruses as a vector.

Success so far

Gene therapy has already been used to treat several genetic disorders. Hemophilia, thalassemia and leukemia are examples of such diseases. Though it has shown promising outcomes, there are some pitfalls that require further development.

The field of medicine continues to improve with the development of science and technology. Gene therapy is one aspect of developing medical techniques that gives hope for many patients with genetic disorders that were thought to be incurable.