Gene therapy is an approach employed by scientists to correct defective genes. Its principles are outlined by genetics, in which genes have a significant role in the human body's response to nutrition and medicines, and contraction of some diseases having genetic component.
The genes are the basic functional and physical units of heredity, and which are carried on the chromosomes. They contain codes of instructions about making proteins as they are the particular sequences of bases. While the genes are more popular to our hearing, it is actually the proteins which are exerting much of the effort in most of the function of life, and constituting majority of the cellular structures.
Genetic disorders are most likely to result should the genes are altered so that the encoded proteins cannot perform their normal functions. In correcting faulty genes, scientists have a few approaches in using gene therapy.
The most common methodology is inserting a normal gene into a non-particular location within the genome, in order to replace a non-functional gene. A vector, a carrier molecule is used to transmit the therapeutic gene to the target cells of the patient. In the present time, the genetically altered virus is the most common vector to carry the normal human DNA. The viruses have evolved a method of encasing and sending genes to human cells in a pathogenic approach.
To demonstrate, the viral vector goes to the target cells which can be the lung or liver cells, and infects the area. The vector will unload the genetic material with the therapeutic human gene into the liver or lung cell. The functional protein product generated from the therapeutic gene is deemed to restore the target cell into its normal state.
A few of the viruses used by researchers as gene therapy vectors include the herpes simplex, adeno-associated, adenoviruses, and retroviruses.
The herpes simplex virus is a class of viruses with double strands of DNA. It can infect a specific type of cell, the neurons. The herpes simplex virus type 1 is a human pathogen that brings cold sores.
Having a single-stranded DNA is what makes the adeno-associated virus. It can insert its genetic material at a particular site on chromosome 19.
Also having double strands of DNA genome are the adenoviruses. Some of the infections they can cause are respiratory, eye and intestinal.
The retroviruses are capable of creating double-stranded DNA copies of their RNA genomes, which can be incorporated into the chromosomes of host cells. An example of it is the human immunodeficiency virus or HIV.
Gene therapy could also be done by altering the regulation of a specific gene, or swapping the defective gene for a normal gene by homologous recombination.
Another way is to repair the faulty gene by way of selective reverse mutation, and thus, allow the gene to its normal function.
There is also a non-viral gene therapy with direct introduction of therapeutic DNA into the targeted cells as the simplest approach. However, this approach is limited because it requires tremendous amount of DNA and can only be employed with specific tissues.
Creating an artificial lipid sphere with an aqueous core is another non-viral approach to gene therapy. Using the liposome, the genetic material can be delivered to the target cell's membrane.
The therapeutic DNA can also be delivered into the target cells by linking chemically the DNA to a molecule that binds to special cell receptors. Once bound to the receptors, the therapeutic DNA are enclosed by the cell membrane and delivered into the interior part of the target cell.
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