How Does Gene Therapy Work?

Gene therapy seeks to replace or repair an abnormal or faulty gene with an introduction of a good gene into the affected area

. The ultimate objective of such therapy is treatment of terminal diseases which are currently difficult to cure. Because it is ineffective to directly inject the good gene by itself into the target cell, a vector or carrier is needed.

There are a few ways to insert a good DNA inside the human body, with some of them less effective. Researchers can use vectors which are viruses that are genetically engineered so as not to affect the patient except to deliver the genetic material into the target cell. The use of vectors can be done intravenously or be injected into the patient.

Another way is to link chemically the good DNA to a molecule so to bind with the special cell receptors. As soon as the good DNA is bound to the receptors, the cell membrane will encompass them and pass through the interior portion of the target cell. This type of gene therapy however is less effective.

Also, an artificial lipid sphere can be created with an aqueous core and task the liposome to carry the good genetic material and pass it through the cell membrane of the target cell.

Currently, researchers are working on employing the 47th chromosome to be introduced into the target cells. Such chromosome would autonomously exist with the regular 46 chromosome, without affecting the functions of the regular chromosomes and without causing any mutations. In order to deliver the good DNA into the target cell, a large amount of vector should be employed, which really, posts some challenges among the researchers as how could they deliver a tremendous amount of molecule into the nucleus of the target cell.

The use of the viruses as the vector has the most promising effects. These viruses are altered so that when they get contact with the cells of the human, they cannot cause diseases. There are two ways to use virus as the vector, namely injecting it directly into the body, and by using an intravenous into a particular tissue in the body, where the individual cells will engulf them.

The intravenous approach of viral gene therapy is done by taking a sample of the patient's cells such as the bone marrow or blood, and the good gene is added into the cells by exposing the bone marrow with the virus. It is then injected back into the bloodstream of the patient.

There are a few viruses used as vector. The retroviruses, such as the HIV, incorporate their genetic material and the new good gene into a chromosome in the human cell of the patient. Retroviral gene therapy has been used to treat SCID or severe combined immunodeficiency.

The adenoviruses, responsible for common colds can deliver the good gene into the nucleus of the cell but are not incorporated into a chromosome. Although they cannot cause common colds, they can trigger an immune response from the body, and may put the patient into further diseases.

As an alternative, the adeno-associated virus, which is derived by adding an innocuous DNA from form adenovirus during the preparation, is used.

by: Charles Godbout

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