Gene Therapy

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Last updated: October 12, 2019

Gene Therapy against Cancer

Cancer destroys lives; turns children into orphans; and brings utter devastation to the lives of victims and their families. After watching my uncle die of lung cancer in 2010, I decided to pursue a degree in biotechnology. I want my life’s work to focus on preventing this type of pain and devastation in others. In my first year in university, my mother was diagnosed with ovarian cancer. During her treatment, she experienced hair loss, losing of appetite, bones pain, nausea, and insomnia. As much as these things hurt me inside as much as it gave me a power and motivated me to obtain a graduate degree in gene therapy.

What is gene therapy?

Gene therapy is a medical practice that involves the transplantation of normal and superior genes into body cells that do not have them or have defective genes in place. A gene is defined as a specific sequence of nucleotides that form the chromosome. They are considered as the basic unit for heredity. Therefore, they determine the characteristic that the offspring will inherit from the parents. However, sometimes the offsprings inherit genetic defects that lead to defective disorders and it is at this instance that gene therapy steps in. This medical practice mainly aims to treat defective disorders by using different approaches.

For instance, a normal gene may be inserted into a genome in the body cells to replace a dysfunctional one or a disease-causing gene may be removed from a normal gene through the process of homologous recombination (Kelly, 90).

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History of Gene Therapy and Development

Gene therapy is very rich in history and it dates back to the early 1970s when the first attempt to perform gene therapy took place. In 1972, Dr.

Friedmann along side his colleague Richard Roblin published a paper on science that forever changed the face of medicine. The science paper suggested that new genes could be introduced into a person’s body cell using viruses. This implied that normal genes could be used to restore missing genes in body cells and to replace defective ones and in turn curing genetic diseases. This was considered as a major breakthrough in the field by many and it contributed to its further development. In 1992, Doctor Claudio Bordignon from Italy performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver normal genes into defective cells. Later in 2002, his continued research led to the publication of the first successful gene therapy treatment for a genetic disorder called adenosine deaminase deficiency (SCID).

In the same year, Researchers working at Case Western Reserve University were able to create tiny liposomes that could be used to carry therapeutic genes through pores in the nuclear membrane and on March 18, 2002, sickle cell disease was successfully cured in mice (Giacca, 51). In 2003, there was a discovery that forever changed gene therapy treatment. A group of scientists in the University of California successfully inserted genes into the brain.

They employed the use of liposomes coated in a polymer called polyethylene glycol (PEG). This was considered a big achievement because there was no method present to insert genes into the brain through the blood-brain barrier. This was mainly because viral vectors were too big to be inserted across the barrier. Some were very optimistic because this achievement had a high potential of enabling the cure of genetic disorders associated with the brain like the Parkinson’s disease.

Additionally, in 2006 Scientists at the Bethesda’s National institute of health successfully treated metastatic melanoma using killer T cells that had been genetically engineered to eradicate the cancerous cells. This was a major development in the field of gene therapy treatment because it signified a new era of effective cancer treatment. In the same year, an international group of scientists attained similar progress by demonstrating the curing capabilities of gene therapy in myeloid system diseases. The scientists announced the successful treatment of two patients who were suffering from a genetic disorder the affects the myeloid cells. In the same year, scientists also came up with ways to prevent the immune system from rejecting a new gene.

This was a major problem before because the immune system would consider the new gene as foreign and as a result, it would reject the cells that were carrying it. This breakthrough was important because it aided in the treatment of diseases such as hemophilia. Recently in 2009, researchers succeeded at curing a brain related genetic disorder called adrenoleukodystrophy using a viral vector that was derived from HIV to inject the new gene into the body cells (Friedmann, 66).

Examples of Disorder Treated By Gene Therapy

Gene therapy has been used in the past years to treat Genetic disorders since the 1970s to the present time. A good example is cancer.

Through the successful genetic engineering of immune cells called lymphocytes, cancerous cells can be targeted in patients with metastatic melanoma. Gene therapy can also be use in the treatment of genetic disorders that target the myeloid cells for instance acute myeloid leukemia Furthermore, because of the positive results in the use of liposomes instead of viral vectors to inject genes across the brain-blood boundary, gene therapy has enabled the successful treatment of disorders such as the Parkinson’s disease. Furthermore, a new development in gene therapy that involves the repair of the messenger RNA present in defective genes has enabled the treatment of blood disorders such as thalassaemia and cystic fibrosis and the cure of other cancer related disorders.

Types of gene therapy

Gene therapy can be classified into two types namely; germline gene therapy and somatic gene therapy. In Germ line gene therapy, functional genes are introduced into the genomes of germ cells, like sperms or eggs. This type of genetic therapy ensures that the beneficial changes due to therapy are passed on to the next generation. It also ensures that genetic disorders and diseases are not passed on to the next generation. The Somatic gene therapy on the other hand, involves the transfer of somatic cells into the patient’s body cells.

The main difference between the two is the fact that in Somatic gene therapy, the beneficial effects are restricted to the patient alone (August, 45).

Gene Therapy Conclusion

In conclusion, aside from the positive effects felt due to genetic therapy, the mechanics involved in the biology are still ambiguous and the further development of this field is required. Certain aspects of the therapy like the reaction of the immune system and the choice of viral vectors needs to be considered to ensure the safety of the patients. Furthermore, the public policy debate can be considered as a factor that hinders its development. The debate is proposed by professionals who mainly feel that the practice of gene therapy on human subjects should be prohibited since the field is still relatively unknown and its developments are premature. Works Cited August, J T.

Gene Therapy. San Diego: Academic Press, 1997. Internet resource. Friedmann, Theodore. Gene Therapy: Fact and Fiction in Biology’s New Approaches to Disease. Cold Spring Harbor, N.

Y.: Cold Spring Harbor Laboratory, 1994. Print. Giacca, Mauro. Gene Therapy. Dordrecht: Springer, 2010. Print. Kelly, Evelyn B.

Gene Therapy. Westport, Conn: Greenwood Press, 2007. Print.

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