Introduction to Gene Therapy

Gene therapy can be simply defined as the treating of serious illness by transferring DNA/RNA to somatic cells. It is also referred to as nucleic-acid-based treatment. Somatic Gene Therapy involves introducing new genes into the somatic cells (i.e. the body).

Future generations of the patients are not going to be affected after he or she undergoes this therapy. When the therapy involves genetic alterations or modification in human germ line as is the case in germ cell therapy, the alterations would be transmitted to offspring. The latter is illegal in all countries as it is considered unethical.

Fundamental Knowledge of the workings of the human body, particularly the functions of a cell, is necessary in order to comprehend the process involve in Gene Therapy. This background knowledge will help dispel whatever misunderstandings one may consciously or unconsciously hold so that the subject under consideration will be understandable to even the layman and as many with the desire to pursue further studies in this field.

The principal task in genetic therapy is the effective transfer of gene into cells. The safety of a transfer agent of a gene must be ensured, its DNA load which is introduced into a sufficient amount of cells should be enough to generate a biological response and act as a go-between in the achievement of the desired healthy gene without a relapse to the former unhealthy state. Vectors that meet criteria like the above are not easy to identify.

Pathologic condition is one area of Gene Therapy is often put to use because of the existence of cells of a particular system or organ whose functions are impaired by reason of the lack of an essential protein.

To get the malfunctioning protein replaced, an alteration has to be made on a gene transfer vector to ensure that it has the gene that produces this protein. Disabled adeno associated virus is an example of gene transfer vectors. The patient is then administered the modified gene transfer agent which enters the unhealthy cells. It is the duty of the vector to transfer the healthy gene to the affected cell and the machinery at work in the cell, turns into the needed protein, the therapeutic gene which eventually fixes the faulty cell.

More information on the most widely employed gene transfer systems need to be assessed and studied in order to better appreciate the important roles perform by gene transfer vectors in Gene Therapy. This information is particularly beneficial to those not verse in the field, although having some understanding of biology, and those with the desire for an in-depth knowledge of Gene Therapy.