Gene Therapy

Zameena Jaffer, 10B

The Impact of Science on Gene Therapy

Science is the modern world's most essential asset. Most of our lives today revolve around science and technology, and without either, we would not be able to function in today's world. For instance, science has transformed old-age farming methods into high-tech genetically modifying methods that are greatly assisting today's farmers. Science has also made reproduction for women suffering from infertility and birth complications possible by providing aid in the form of 'test tube babies' and surrogacy. Another major step that science has taken comes in the form of Gene Therapy. 'Replacement' and 'modification' are the most commonly used terms associated with Gene Therapy. Defective genes are either replaced, added to, or modified using DNA injections, or genetic engineering techniques to correct segments of DNA and genetic material so that it may act as a cure for patients that have defective genes or inherited defects. Gene Therapy allows scientists to medically replace defective genes, or medically add to normal genes to living cells in the hopes of correcting or remedying a genetic defect. Gene therapy allows doctors, with the help of scientists, to cure genetically caused conditions by the insertion of corrective genes (Clay Farris). There are plenty of advantages that come from the use of gene therapy which will prove to be extremely useful in the future, especially because the diagnosis of new life-threatening diseases is increasing rapidly. However, gene therapy does not only provide advantages. Like most scientific methods, gene therapy can also reject our bodies when inserted, causing harm and even death. Gene therapy can also cause environmental hazards, human health risks and even economic concerns.

The process by which genes are 'used as medicine' is entirely based on science. This process involves the transfer of working or therapeutic genes into specified cells of an individual in order to attempt to repair a faulty gene that is usually a result of a genetic defect (Centre for Genetics Education). There are two traditional methods in which doctors, with the help of scientists, perform gene therapy. The methods that the doctors used are generalized into two gene therapy techniques known as 'ex vivo' and 'in vivo' gene therapy techniques.

The ex vivo gene therapy technique consists of those methods that require the cells from the affected tissue area in the patient’s body to be surgically removed. The doctors then inject or splice (merge) the new DNA that will correct the disease into the affected cells and allow the cells to divide. Once the divisions have occurred, the new and improved tissues are inserted back into the affected area in the patient’s body. In order to carry out these type of techniques, the doctors only require to culture the patient’s bone marrow. This is due to the fact that the bone marrow produces the blood that will travel, eventually, throughout the body – thus eventually reaching the affected area as well. Although the ex vivo technique is simpler, it is also more painful than the in vivo technique and usually requires the patient to undergo surgery twice. The reason behind the dual surgery is that the first surgery is conducted in order to extract the marrow and the second is conducted in order to replace the marrow back to it’s original position. The culturing of the DNA may also take many hours to complete ().

On the other hand, the in vivo gene therapy technique consists of those methods

that do not require anaesthesia or even surgery. This technique includes the therapeutic DNA being injected directly into the body cells of the patient. This is done vie one of two types of viruses. The most commonly used and the simplest virus used for this technique is a known as a retrovirus. The characteristics of the retrovirus greatly affect its success rate. The perfect retrovirus was created by a doctor named Richard Mulligan of MIT. He synthetically created a retrovirus that existed solely to deliver therapeutic DNA during gene therapy and had no reproduction sequence. The retrovirus contained only the new DNA that had been spliced into it and did not contain any viral DNA that could possibly make the patient sick. The remarkable thing about this technique is that the retrovirus dies after injecting the diseased and affected cell with the new therapeutic DNA. Retroviruses are a safe and effective vector to use for gene therapy as it provides long-lasting effects. This is the more complicated of the two techniques, but also the more effective technique. Although this retrovirus seems to only provide advantages, it does have some limitations. The new DNA injected into the patient’s body can only help the new daughter cells that the body creates, and not the daughter cells that already exist within the body. ().

Another type of virus is known as the adenovirus and it too is used for the in vivo technique and it is equivalent to the common cold virus. However, the adenovirus does have some side effects. Patients who chose to use the adenovirus as a vector for their gene therapy will suffer from a runny nose and a temporary sore throat. Although the adenovirus works in similar ways to the retrovirus - it too dies after being injecting its spliced therapeutic DNA, it has a hand over the retrovirus because its effects are much more immediate. Also, scientists and ...

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Through these gene therapy techniques are born methods or forms of transferring genes through gene therapy. There are two most commonly used transfer gene therapy forms in today's scientific world.

The first gene therapy form is "Somatic Therapy". In this method, doctors and scientists insert genes into the tissues of a living person. After this has been done, the inserted gene will begin to be expressed through the modified cells in the individuals body, creating a new phenotype which will later aid the patient. However, before this process can take place, the doctors have to determine what gene to insert that will aid the patient and how to go about inserting that particular gene. Decisions of which is the appropriate gene to introduce into an individuals body is obvious for some diseases, but this is not always the case due to the fact that other gene decisions for uncommon diseases require more ingenuity. For example, AIDs researches have found, through investigation, dozens of genes that make cells resistant or inhospitable to HIV infection ().

The second gene therapy form is "Germ-Line Therapy". This from of genetic therapy involves the scientists or doctors modifying germ cells (gametes) which will go on to pass the change onto the next generation of offspring (the patients’ child). This procedure spares the child from certain genetic problems that would have otherwise occurred because it corrects the genes in the egg or the sperm that the patient will use to conceive. Every cell in the offspring will possess the transplanted gene due to the fact that every cell descends from a fertilized egg. Scientists feel as though Germ Line Therapy is a far more effective from of transferring genes as opposed to Somatic Cell Therapy due to the fact that during Somatic Cell Therapy, the transferred genes only enter a small portion of the person’s cells and can, eventually, even stop functioning ().

Due to the fact that gene therapy procedures are constantly evolving, there are numerous amounts of various techniques that were tested and approved, however, there were also times when some techniques were disapproved and even considered dangerous.

For example, one technique includes a therapeutic DNA into a cultured endothelium (the membrane that lines all the blood vessels) and then splicing or grafting it into a patient. Another technique includes a patient being submerged into a solution of therapeutic DNA and then receiving an electrical shock so that the patient’s skin pores open up and allow the DNA solution to enter. Other techniques also include injecting the liver with the therapeutic DNA, skin grafts and, also, connective tissue grafts.

Gene Therapy have both advantages and disadvantages. These altered genes solve numerous medical problems. Firstly, due to the fact that the field is advancing so rapidly, scientists are introducing treatments to terminal diseases that doctors would have previously thought to be hopeless. Secondly, gene therapy has triggered the production of medicines in mass quantities which has resulted in purer and cheaper medicines. Gene Technology that is associated with genetic engineering that is used to modify genes and create medicines runs many large factories and employs thousands of workers. Therefore, without genetic engineering and without gene therapy, interest rates and medicine prices would go up and there would be a terrible loss of jobs. Not only does genetic engineering and gene therapy employ a lot of people, but it also can decrease death rates through somatic therapy and immortality rates through germ-line therapy. With increasing diagnosis of life threatening diseases, medicine and surgery prices would decrease and become even more affordable. Some people feel as though anything gene-therapy associated is trying to give someone that is born with genetic deficiency or develops a terminal disease, like cancer, a chance at a normal and healthy life – and that outweighs all the disadvantages as this is a miracle all in itself. However, not everyone feels the same way because although gene therapy may seem like a gift from science, the risks involved in gene therapy are just as dominant as its benefits.

After much analysis of the techniques used to perform gene therapy, many critics have come to realize that though gene therapy helps save lives, it still hits a road block from time to time when the therapies limitations are revealed. However evolving the techniques of gene therapy are, they still face difficulties. One of the techniques that faces a limitation is the technique of identifying the genetic fault in the patient’s body. Approximately 2% of all illnesses (Huntingdon’s disease or cystic fibrosis for example) are directly linked to a single faulty gene. This fact makes it extremely difficult for scientists and doctors alone to figure out exactly what the genetic defect is and if it even has anything at all to do with a genetic component that they can modify. The next technique that faces a limitation is delivering the new genetic material to a patient’s cell and keeping it working. Gene therapy is continuously burdened by the difficulty of inserting genes into cells. At the moment, the most commonly used inserting methods are viruses. These include adenoviruses, lentiviruses, retroviruses and adeno-associated viruses. These viruses are used as ‘vectors’ to introduce the new, and modified, genetic material into the patient’s body. The problem, however, is that vectors may trigger an immune reaction in the patient’s body which would cause it to reject the inserted genetic material rendering it useless and ineffective. One of the biggest challenges that remains for gene therapy is the search for a reliable vector.

Not only does gene therapy face only technical difficulties, but after much ethical evaluation and analysis of gene therapy, skeptics have come to a conclusion that there is a likelihood of gene therapy causing harm, and thus skeptics are encouraging the world to weigh the risks of gene therapy against its probable benefits. There is only so much science can do to keep the technology of genetic modification under control and under full surveillance. For instance, the genetic screening needed to see what disease is present is raising many ethical and legal problems and producing much controversy as a result. People believe that prenatal tests could lead to a possible increase in the abortion rate. The tests are seen as a problem once again as those people that test positive for a defect gene have a hard time trying to find an adequate insurance service to cover their expensive treatments as they are tagged by the insurance companies as a high risk – which they wouldn’t have been tagged if they had never tested. Also, many couples have found out that they are carriers of a disease and now have been faced with the dilemma of deciding weather of not they want to have a child that has a high probability of inheriting that genetic disease. Another pressing problem is the actual of gene therapy. If scientists were able to identify the gene that contributes to beauty or youth, then the techniques of gene therapy would be manipulated due to the fact that our society at present day are obsessed with the idea of beauty and youth. After being manipulated, the techniques would later be monopolized by the cosmetic industry as a technique to enhance beauty or to “turn back the clock.” It is up to federal regulation to decide which is a more important genetic problem – a large nose or cystic fibrosis?

Although one might think that the subject of gene therapy is one for doctors and scientists, economists do seem to get involved from time to time. The fact that people these days are concentrating more on the profitability of gene therapy rather than the fact that it can cure life threatening diseases has serious consequences. The percentage of people diagnosed with ‘minority diseases’ or serious single gene disorders such as cystic fibrosis or Huntington’s disease is very small. This fact makes research in the area of such diseases commercially unattractive. This also means that due to the profit motive, the interests of the rick will drive the exploitation of technology into fields such as cosmetics. This is proved by the ever-developing fears that gene therapy is being used and misused in sport. To make matters worse, people also fear that gene therapy is being misused to make ‘improvements’ to peoples appearances and even make ‘designer babies’.

Though there are countless debates on whether or not gene therapy has any environmental implications, analysts have proven that though gene therapy helps save lives, it does do some harm to our environment in the process. Due to the fact that the medicine products used during gene therapy are chemically derived and then transmitted into a patients, that transmission can have ‘shedding’ side effects from the patient to third party persons, animals, plants or the environmental as a whole. The residue and chemicals from the transmission of genes through gene therapy, may infect the environment and can prospectively disturb the balance of nature. Scientists predict that a worst case scenario would result in a high-cost and can threaten the ecosystem. Due to the fact that this is chemical pollution, however, there is no clean up or reconciling for the genetic pollution that gene therapy may cause.

Being such big players, the molecular biologists, bio technicians, scientists and genetic engineers that invented gene therapy are bound to be seized into the political world. At the moment, genetic engineering is ahead in the game due to the mass production of therapeutic gene solutions and medications. Politics reveals that opponents of current genetic engineering are claiming that there has been a massive 'power shift' that leans towards biotechnology due to the increasing number of therapeutic gene solutions and medications produced each year. Analysts fear that genetic engineering is gaining too much control over production and the scientists that buy their products. Bio and gene technology is, without a doubt, dominating the medical production market. The producers of therapeutic gene solutions and medications are trying to silence their opponents by claiming that, if given the chance and allowed dominance, their products have the innovation and the capability and capacity to prevent. Opponents and skeptics think that genetic engineers are biting of more than they can chew .They also believe that genetic engineers are committing biopiracy due to the fact that they are committing foreign exploitation of natural resources and chemicals in order to produce their solutions and medications.

Many ethical questions have been raised concerning new gene therapy technology. Now that scientists have proved that they are able to replace, add to, or modify defective genes using DNA injections, or genetic engineering techniques to correct segments of DNA and genetic material so that it may act as a cure for patients that have defective genes or inherited defects. The world fears that this gene therapy technology cannot be trusted in human hands. They are suspicious that this new technology will be used as an instrument for the manipulation of human genetics. They feel as though sciences well-meaning actions in creating gene therapy will take a toll for the worse and come to an objectionable end and damage human life as an unforeseen consequence. The world is afraid that ethics will not be respected and that there cannot limit the damage if such a great power is placed in human hands. Humans are still not intellectual enough or innocent enough to be trusted. They fear that doctors and scientists will get overly confident with the gene therapy technology and not weigh the risks and benefits of gene therapy for each individual. There is a risk of infection by viral vectors with current gene technology, and even causing predisposition to cancer. Also, the world feels as though somatic cell gene therapy is ethically acceptable whereas germ line therapy is not as they feel that those conducting germ line therapy are attempting to be God. They feel as though germ line therapy should be studied for any potential adverse effects before it is clinically tested on human beings. Other cultural groups feel as though scientists have no right to genetically alter nature, especially if done for only profit and convenience. Cultural groups feel as though germ-line therapy should be eliminated as an option completely as they fear that doctors will get influenced by the cosmetics world and begin altering human attributes. They fear this because the alteration of human attributes can, ultimately, lead to inbreeding problems, gender imbalances and reduction in genetic diversity.

Gene therapy has given hope for treatments for previously untreatable diseases, and can also prevent certain diseases through correction processes of genetic disorders. However, gene therapy is clearly under development and is still clearly a work in progress due to the fact that, in the short to medium term, gene therapy will not be used to prevent diseases exactly, but it will be used to develop ‘gene-based’ treatments for terminal diseases, such as AIDs and cancer, that will work more effectively. It is no surprise that gene therapy is considered as a major breakthrough in medical science, but the world must not forget that, like every penny as two sides, it also has its potential for abuse, limitations, and commercial imperatives. Scientists and doctors alike must remember that is the human ‘need’ that is driving the progress of gene therapy and not the human ‘want’. However, placing genes as the emphasis to determine health and disease will ultimately lead to suffering as the underlying causes will be neglected. The main aim to not restrict the society from being overcome by ‘genetic determinism’ or ‘genetic thinking’ and to keep them educated on the fact that they must try avoiding the pitfalls that they will face concerning gene therapy, while enjoying its benefits as well. Gene therapy has promised the world many benefits, no doubt, and they have definitely delivered their Although genetic therapy promises the world a lot of benefits, scientists still have yet to conduct in depth research and need to assess the potential risks that genetic therapy can cause. Not until scientists conduct studies and surveys to prove that genetic therapy is not a threat to human beings health and the worlds ecosystems, will the debate over the release of gene therapy ethics cease to exist. Despite the fact that gene therapy has a number of inviting benefits such as providing a cheaper substitute of medicine due to mass production, it is still debatable that living organisms like human beings are complex and thus tampering with such life forms can have unintentional effects. Fortunately, the worlds common interest is being supported by concerned scientists organizations who demand mandatory gene therapy trials before any permanent procedure is conducted. Just remember: a penny has two sides. Now it is up to you to determine heads or tails.

The Impact of Science on Gene Therapy