Gene Technology: Gene Therapy

Gene Technology: Gene Therapy
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Gene Technology: Gene Therapy
1 Biological Basis of Gene Therapy
1 Description of the technology and how it works
The purpose of gene therapy is to introduce a gene into cells to either replace the defective genes or synthesize the intended functional protein (Mammen, 2007). It is a technique used to introduce a normal copy of a gene for protein function restoration, particularly if a mutation of the defective gene results in faulty or missing protein. To perform gene therapy, scientists insert genes directly into cells which have lost their functions. Genetically engineered vectors such as non-pathogenic viruses act as carriers to deliver the normal genes into cells by infecting them (U.S. National Library of Medicine, 2014). Viruses, such as retroviruses, are usually engineered to make them non-virulent in human cells. The viruses integrate their genetic material and the new gene to be transferred with the human chromosome in the target cell. However, in adenoviruses, the viral DNA is introduced into the cell nucleus without integrating the DNA into the human chromosome.
1.2 How the technology works
Vectors carrying the desired gene can be administered intravenously into the target tissues and taken up by specific cells. Alternatively, cells can be extracted from the patient and then exposed to the vectors in the lab (U.S. National Library of Medicine, 2014). The cells that have been infected by the vectors are then injected into the cells of the patient. Effective treatment of the patient will be demonstrated when the new gene successfully delivered by the vector will synthesize a functional protein as shown in Fig 1. During the process of gene therapy, scientists encounter several hurdles which they must overcome if the technique is to be successful in the treatment of diseases. For instance, better ways of gene delivery and specificity to target cells has to be sought if this process is to be effective. In addition, the new genes introduced into cells have to be controlled precisely in the human body/patient.
Fig 1. Gene therapy using adenovirus (U.S. National Library of Medicine, 2014).
1 What gene therapy accomplishes
Still in its experimental phase, the technology uses genes to either prevent or treat diseases. It is hoped that doctors will be able to treat diseases by inserting genes into the cells of patients instead of using drugs or performing any surgical operation. The technique accomplishes the intended purpose by introducing a novel gene which helps the body to fight an infection, knocking out or inactivating the mutated gene that expresses a nonfunctioning protein, or by replacing a mutated gene which causes the infection with a healthy one (Mammen et al, 2007). Gene therapy has the potential to treat a variety of diseases such as cancers, viral infections, and genetic diseases. However, since the technique is considered to be risky, the effectiveness and safety of gene therapy can only be tested for the treatment of infections that currently lack cures.
2 Scientific principles making the gene therapy possible
The genes, which are contained in chromosomes, are the basic unit of heredity. These...