Physical Science

Genetic

Germ line gene therapy is a form of analysis involving the alteration of germ cells by introducing well-designed genes and incorporating them into the cell’s genomes. Germ cells include spermatozoa and ova that combine in the process of fertilization. The result is a zygote that eventually grows and alters into a mammal. These cells can be modified in the initial stages of their modification, including during the first few hours of fertilization and before the embryo makes its way into the uterus. Alterations to the cells will result in changes in the outcome of the offspring, whether physically notable or not. As a result, consequent generations will also be affected. This form of therapy is aimed at reducing the risk of repetitive diseases, especially those that are transmissible or genetically hereditary in nature.

Somatic gene therapy involves the alteration of somatic cells by the use of therapeutic genes. The word “somatic” is coined from the Greek word ‘sōma’ which represents ‘body’. These cells are found in places where the germ cells are not present. Examples of such locations include connective tissues, blood, bones, skins and most internal organs (Johnson & Library of Congress, 2004). Alterations to the patient’s body will not be transmitted to future generations making this procedure purely a one-person deal.

A fundamental constituent of genes is DNA that comprises of elongated molecular sequences known as nucleotides that store genetic information. DNA is therefore responsible for the management of the formation of a cell’s constituent substances, which are contained in stretches known as genes. There are similarities that would be notable when scrutinizing DNA of a gene altered in either somatic or germ line therapy (Johnson & Library of Congress, 2004). Disparities that are manually created by adding diverse sequences next to the gene would lead to controllable treatments. The main difference in the two kinds of therapy is the type of cell that is viable for the operation with foreign DNA.

Somatic cell therapy can be utilized when amending genetic defects that lead to ADA or PNP enzyme deficiencies. These result from an anomaly or the enzyme known as adenosine deaminase or a deficiency of the same. Such anomalies in the DNA of the host result from mutation that is characterized by the formation of erroneous sequences such as the misplacement of a nucleotide due to an addition or omission of another. As a result, a functionless enzyme is formed and thus a normal protein is usually missing. Reactions controlled by ADA are therefore affected.

ADA usually leads to the breakdown of chemicals, and because of the anomalies described above lead to accumulation of such chemicals and the displacement of the byproducts of this process. Such chemicals are toxic and the cell cannot function properly when fighting various immune-deficiency syndromes. This is where somatic gene therapy comes in, and it usually involves retrieving bone marrow and adding normal genes of the deficient enzymes into this marrow (Johnson & Library of Congress, 2004). The altered cells are reinserted into the body where they replicate and, if successful, restore immune function by creating the missing enzyme to breakdown toxic chemicals.

This form of therapy achieved by manipulating the Germ cell gene is still theoretical, at least as concerns human use. On the other hand, somatic cell therapy has been tested on humans. Germ cell gene therapy has the potential to eliminate problems on a larger scale, especially since its effects stretch to future generations. However, it does not come with a full assurance of complete success, as there is always a possibility of unforeseen mutations or the resultant effects of unexpected gene modification. Animal testing can be used as an avenue of testing the efficiency of the process and an analysis of any resultant anomalies should be noted. However, there is always the risk of causing problems in otherwise healthy embryos thus leading to illnesses or death of the offspring.

References:

 Johnson, A. J., & Library of Congress (2004). Human gene therapy. Washington, DC: Major Issues System, Congressional Research Service, Library of Congress.

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