Pro Germ-Line Gene Therapy

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Gene therapy is a highly controversial topic and there are reasons why people are either for or against it. All of us carry defective genes and may not know it. Gene therapy is a route to tackle the fundamental biological cause of the disorder: the faulty gene being replaced by a healthy gene. Genes are the fundamental physical and functional unit of heredity. Each genetic makeup of a person is unique and this is what makes us different from one another. A mutation in any one of these genes can result in a disease, physical disability or shortened life span. These mutations can be passed from one generation to another. With germ-line gene therapy, the inherited diseases or physical conditions due to these mutations could be eliminated. Germ-line therapy should be practiced despite the ethical claims, because it is a long-term solution to save the future generations from dealing with the defective gene pool.

Gene therapy is an experimental form of treatment that uses gene transfer into the cell of a patient to cure the disease. The two types of gene therapy are somatic cell gene therapy and germ-line gene therapy. To differentiate these two gene therapies, somatic cells do not transmit genetic information to succeeding generations, while germ-line cells provide genetic information to offspring, as well as to the future generations descended from those offspring. Gene therapies can work by replacing a disease-causing gene with a healthy copy of the gene and inactivating a disease-causing gene that is not functioning properly. The idea is to modify the genetic information

of the cell of the patient that is responsible for a disease, and then return that cell to normal conditions.

According to the Food and Drug Administration, the definition of gene therapy is “to modify or manipulate the expression of a gene, to alter the biological properties or to alter the biological properties of living cells for therapeutic use. (FDA, 2021).” Somatic cell gene therapy is ethically less controversial of the two therapies, because the treated defective gene does not pass down to the future generations. It is safe for management of disorders in human beings. Any modifications and effects are restricted to the subject and not all the future generations. That is why somatic cell gene therapy does not raise ethical concerns.

Although somatic cell gene therapy is a good treatment for specific genetic defects, one disadvantage of using this type of gene therapy is repeating the treatment for every future generation. According to Leroy Walters and Julie Gage Palmer in an article called Germ-Line Gene Therapy, the disadvantage of somatic cell therapy would be prolonging a defect. Examples used in the article were Sickle cell and cystic fibrosis and the article states:

If somatic gene therapy is employed in significant numbers of people afflicted with recessive genetic diseases, some of those people’s somatic cells will be

able to function normally, but their reproductive cells will remain unchanged, thus assuring they will be carriers of genetic disease to the next generation.”

(Palmer, 637)

Using somatic gene therapy is beneficial in the short term and the benefits may not last beyond one generation, because the treatment has to be reapplied. The removal of the defective gene does not treat all future generations. Somatic cell gene therapy is a temporary relief, while

germ-line gene therapy has a promise to permanently remove the gene disorder safely and effectively in every future generation. The benefits of germ-line gene therapy far much outweigh the ethical concerns.

Ethicists consider germ-line gene therapy inappropriate, because it would violate the rights of the future generations to inherit an altered gene. Germ-line gene therapy has the potential for preventing inherited diseases. However, this approach is controversial because it does not simply affect the patient, but impacts the descendants of the patient. If all specific dominant genetic disorder were to be eliminated, that single dominant gene would also disappear.

Germ-line gene therapy has the potential to wipe out genetic disorders before they start and eliminate pain and suffering for the future generation. The widespread use of germ-line gene therapy to eradicate disease genes means that any beneficial traits associated with those particular genes would also disappear. It may be applied to prevent defects and ensure that the root cause of disorder is removed, rather than only easing the symptoms. It will enable the patients to be free of the defective gene and the possibility to be cured of the disease completely. The patient will have a chance to enjoy life once the process is successful.

Preventing the gene disorders into the future generations is one of the advantages of using germ-line gene therapy. With the use of germ-line therapy, somatic-cell therapies will no longer be needed and the chance of children and their right to be born healthy without genetic burden will become a reality. With the effective germ-line gene therapy, the cure will permanently eliminate the genetic diseases. Germ-line gene therapy will be more effective than the somatic-cell gene therapy, because its effects are not limited to the actual subjects being treated. All of their offspring will be free from the genetic condition treated with germ-line gene therapy.

Walter and Page mentioned that gap between the rich and the poor will be widened if germ-line gene therapy is approved. The article states, “However, if enhancement interventions were safe and efficacious, the long-term effect of such germ-line intervention would probably be to exacerbate existing differences between the most-well-off and the least-well-off segments of society.” (642). Once germ-line gene therapy proved to be effective, safe, and available, then the health care insurance industry or tax payers would fund the life-saving procedure to make it equitable for all, because it is cheaper in the long run and more people will have access to the treatment. There are possibilities that the health care cost would be saved; lower the probability of carriers and those expressing a defect will reduce the need to repeat the treatments from somatic gene therapy. The advantages outweigh the drawbacks in this case once germ-line gene therapy gets FDA approved, then the treatment will be viable.

In conclusion, germ-line gene therapy holds promise as an effective treatment option for a variety of diseases at some point in the near future. Both somatic and germ-line gene therapy have their own advantages and disadvantages from the amount of treatment to be reapplied as in the case of somatic cell or the permanent solution that affects future generations as in the case of germ-line gene therapy. It enables the generation being treated to lead a healthy life.

Germ-cell gene therapy affects not only the actual persons treated, but also their offspring. Therefore, a single intervention might affect all future generations that share a common genetic heritage with the original patient. Besides, if germ-line gene therapy has the potential to remove a disease completely from the population, it will reduce or remove the long term healthcare costs of treating the disease extend beneficial effects to all. It will eliminate the need for health procedures, because many diseases are lifetime health issues.

Overall, the potential in germ-line gene therapy is greater than somatic cell gene therapy. If society learns to use the abilities of germ-line gene therapy appropriately, then its potentials will be of valuable use by offering patients therapeutic genes to treat, cure, and ultimately prevent a wide range of diseases that now plague mankind.