SCID more commonly known as “Baby In A Bubble” is another disease which can be treated successfully with the use of gene therapy. It is a very rare condition and one that only occurs with boys. SCID “is caused by a single mutated gene, and this means that patients to live in sterile conditions (bubble) or risk picking up a life-threatening infection.” Gene therapy adds to the body, via viral vectors, a correctly functioning version of the mutated faulty gene that causes SCID. This would generate further cells to pass into the blood stream and protect from infection. Below is an image of Rhys Evans who was the first ever child to receive this new treatment.
What Is Cystic Fibrosis?
Gene therapy has also looked into cures for the disorder cystic fibrosis. Cystic fibrosis is a genetic disease that affects cell membranes where a faulty gene does not make the correct protein in the cells. The deadly disease can be passed on through offspring and can be very serious and fatal. Genes are found in the cells of our body called chromosomes. There are normally 46 total or 23 pairs of chromosomes in each cell of our body. The seventh pair of chromosomes contains a gene called the CFTR (cystic fibrosis Tran membrane Regulator) gene. Mutations or malfunctions in this gene are what cause Cystic Fibrosis. The disease can be genetically passed onto to offspring if there are two recessive alleles gained from parents. You can be either a carrier which means you do not suffer from the disease but you can pass it on genetically through offspring OR you can suffer cystic fibrosis and pass it on genetically. There is no specific gender that cystic fibrosis is more common in. Both males and females are capable of getting the disease.
How does a person know if they have cystic fibrosis? Cystic Fibrosis can affect multiple areas of the body. The main areas of the body that the disease affects are the lungs and digestive system. There are many symptoms to this deadly disease. However, A person does not need to have all the symptoms in order to have cystic fibrosis, but they usually show most of them. One of the most noticeable symptoms in all cystic fibrosis patients is the large amount of built up mucus. Cystic Fibrosis is a disorder that causes the body to produce larger amount of mucus than normal. In a normal person, mucus in the lungs helps to get rid of germs and bacteria in the air. “In a Cystic fibrosis patient the lungs become covered with sticky mucus which is hard to remove and causes infection from bacteria. Over time infections cause the lungs to become extremely weak, therefore ending in respiratory failure.” This is why cystic fibrosis patients have an unfortunate life span of 20-30 years. Below are the main symptoms that a person may suffer from having cystic fibrosis:
- Asthma
- Small growths in the nose
- Many psychological affects
- Diabetes
- Liver disease
- Coughing a lot of mucus up
- Smelly bowel
- Young children do not put weight on as fast as normal
- Lung infections
- Fertility problems in women due to thicker mucus making fertilisation more difficult
- Born children with cystic fibrosis develop slower than normal
- Infertility in men
- Salty skin and sweat
- Affects the mucus glands of the lungs, liver, pancreas, intestines and skin
- Enlarged liver and spleen
- Enzymes in the body do not work properly in the body but mainly in the digestive system.
How Can Gene Therapy Be Used To Treat Cystic Fibrosis?
Scientist can deliver the engineered bacterium containing the normal gene in a number of ways. There have been multiple approaches to transfer the gene such as, viruses, liposome’s and capsules. However the most common way to deliver the healthy gene to the lung is via a virus (viral vectors). The gene would be put into the body through the nose or mouth using an aerosol. Once in the body the bacterium holding the correct gene starts mitosis, divides rapidly, making exact copies of itself replicating the plasmid. Due to rapid division, there are soon millions of bacteria, each carrying the CTFR gene. If all goes as planned the gene injected will replace faulty genes and the cells begin to produce the normal CFTR protein and cystic fibrosis will then be terminated. However this is not always the case.
Ideally we would think that transferring the normal CFTR gene into the affected cells would result in CFTR replacing the faulty immediately. However recent studies and statistics have shown that very few cells take up the virus and express the CTFR gene meaning the treatment has little effect. There is no 100% cure for cystic fibrosis at this time gene therapy is still a relatively inefficient treatment. All that can be done to cystic fibrosis patients is reliving them from some the symptoms with drugs or alternative treatments such as physiotherapy so that they can live a relatively good life. There is a long way still to go before we have a cure for cystic fibrosis, but it is clear there is development and we are moving in the right direction.
How Does Bacteria Grow?
Bacteria go through the mitosis process. This is the division of a cell to nucleus to produce two daughter cell nuclei with genetically identical material. This process occurs in order for cell growth. There are four key characteristic phases of the bacterial growth cycles. The first of the four phases is the Lag Phase. Immediately after the bacterium has been placed into a new environment, the population remains temporarily unchanged. There number of cells is not increasing but the cells maybe synthesizing proteins enzymes and nutrients. The next of the four key phases is the Exponential Growth Phase. This phase of growth is where all cells are rapidly dividing by mitosis. At this phase there is no factor limiting the growth of bacteria. The rate of exponential growth increases as time passes. The third phase of the growth cycle is called the Stationary Phase. The growth reaches a peak and a point where death rate equals the birth rate. The population growth of the bacteria is limited from either a change in ph, lack of space, low oxygen levels or the exhaustion of available nutrients. Some bacteria may also produce secondary metabolites during this phase. The 4th and final phase of the bacterial growth cycle is the Death Phase. If the growing conditions continue to stay the same at stationary phase then a death phase is followed. This is where the bacterial cell population is slightly decreased. At this point the nutrients have all been used up and the secondary metabolites begin to kill cells.
Graph Analysis And Explanation: Bacterial Growth Rates
The line graph that is shown here on the right illustrates the growth rate of the bacteria that our company will be using to create cures for genetic disorders. The bacteria that will be used have been given the name X for comparison purposes. As Mentioned previously, these bacteria go through a process called mitosis, which can also be called binary fission. This particular bacterium carries out the cell division process every 60 minutes. To allow maximum results and ensure that the bacteria will be effective our company must have created over 15 million bacteria cells to allow enough protein or chemicals to be produced by the bacteria. If this is the case, we can attempt to successfully create an effective treatment or cure for cystic fibrosis and other genetic disorders. In the graph shown above, we can clearly see that it takes just over 24 and half hours to reach our company’s desired amount of genetically engineered cells of bacteria. You may have also identified that the bacteria numbers double every hour. However as shown in the second graph below, if this bacterium was given a constant supply of nutrients and a correct temperature, the growth rate of the bacteria will increase, thus making it faster to produce the correct number of bacteria.
The second of the two line graphs is shown here and represents the growth rate of bacteria that has had been purposely made more effective. This bacterium has been given the name Y. This bacterium also carries out cell division and mitosis; however this particular bacterium divides into two daughter cells every 30 minutes rather than the 60 minutes of above. This makes it more efficient than Bacteria X. However this comes at quite a cost as it consumes proteins and nutrients unlike Bacteria X. The graph of Bacteria Y growth rate evidently shows that it takes exactly half the amount of time (12,5 hrs) to reach our 15 million bacteria cell mark. You may consider Bacteria Y to be the ideal bacterium to use for our company’s production needs in creating cures for genetic disorders; however before you get excited about this prospect, as I touched on before, using Bacteria Y will come at a huge cost. There would have to be quite a large sum of investment to grow the bacteria faster. The costs would be due to the purchasing of nutrients and proteins for the bacteria to feed on; and on top of this an extraction pump would have to be purchased to take away the toxic secondary metabolic products that could hinder the growth of the bacteria.
Are Data And Results Reliable And Valid?
The reliability is referred to how consistent and similar the measurements are (concordant data). A measurement is said to be reliable if future measurements are seen to be similar in the same situation. In my situation, I know that the data collected was reliable as the numbers collected were from successfully completed experiments and were published in a registered official science guide. Each individual test was repeated over to get concordant data. This would leave results where anomalous results could be ignored. The validity basically refers to whether the facts and figures are correct and whether they are what they claim to be. Validity also stands for fair testing. I ensured that the results were going to be valid as experiments for each influencing factor were carried out in the exact same conditions where only one independent variable was altered at a time. Examples of variables that were controlled in the experiments are, the surrounding temperature, amount of nutrients and proteins given to the bacteria to feed on and whether f the secondary metabolic products were removed using an extraction pump.
What Are The Costs?
As mentioned numerous times throughout this report, the costs that come with implementing, not just this technology but any new technology could be extremely high. One major cost would be the salaries that have to be paid to the scientists. The genetic engineering technique is a complicated one and is one that requires highly skilled and trained scientists who hold a degree. And as we all well know the more able and qualified a person it the more money they are entitled to earn. Secondly, as advances in technologies grow, the prices for equipment and machinery have increased significantly. As gene therapy and genetic engineering requires fairly rare equipment. For example an extraction pump would be necessary to extract the toxic secondary metabolic products that affect bacterial growth. On top of this, some sort of heat chamber with adjustable temperatures would also be needed to increase the yield of bacteria. Therefore the company would also have to cover these costs. In addition, specific proteins and nutrients, which could be expensive, would also need to be purchased for the growth of the bacteria. So as you can see already a serious amount of investment would have to go into the use of this technology.
The developing world, with its high population and high unemployment rate would struggle with the costs the most. They would be far better off using more labour intensive methods of production. If genetic technologies were to be applied in less developed nations, where labour is cheaper than in the developed countries, the price of genetically engineered products could possibly be lowered however the costs of genetic engineered seeds for example, are too high for farmers from developing countries to implement. Not only will farmers lose money, but unemployment, which is already a problem in many developing nations, will increase, as farmers need to hire less labour to produce the food and crops. The profit margin here could potentially be huge; however it also has the potential to be disastrous. As so much money would go into producing genetically engineered products, the price these would be retailed at would be extremely high, therefore it is thought that only the wealthy members of the society would be able to afford it. There are a lot of consumers out there on tight budgets any may not be able to afford the products.
Time and money would also have to be invested into further research into the subject. The company must make sure, for the protection of consumers that any genetically engineered products do not have any side effects. Specialist market researchers and research scientists would have to be employed to ensure that there is absolute maximum knowledge on the technique so that this can be a successful project, and clearly the more people employed the higher the wage bill.
What Are The Ethical, Moral and Social Impacts?
It is quite evident that this new form of therapy has opened up many possibilities in the sector of science and medics. It can lead to many useful developments in the area of medicine, agriculture, industry and conservation. However, gene therapy also has the potential to trigger ethical, moral and social problems. Gene therapy involves the transfer of a gene from one organism to another. This modification would change the physical nature of an organism and this is why gene therapy is such a popular issue of debate and protest. Many people have different opinions and views on this topic for various reasons however there are generally more negative views from the public.
An ethical and moral issue is the human interference with the whole human genome. It could be possible to pick and choose most characteristics for unborn children including their sex, using gene therapy when they are single cells, a fertilised egg. The advantages here with this is that you would be able to dispose of genetic disorders and diseases by replacing faulty genes, but the major concern at the moment is ‘designer babies’. This simply means designing your baby to your specification, for example hair colour, eye colour, sex etc. One problem with this is that it would be a very expensive procedure due to the highly skilled doctors and rare equipment needed. Therefore only the richer members of society could afford it. This could lead to some very large social problems for instance; it could lead to a social underclass. With only wealthy people being able to go through with the expensive procedure, the slightly poorer, may see this as prejudice against society and unfair.
There are also some concerns that the genetic information of each individual could be used by schools when deciding to accept a new pupil; Meaning if that person didn’t have ‘good intelligence genes’ then the school could reject that individual. In addition, with the advances in gene therapy then it may be possible to ‘design’ babies for a specific career, for example making them athletic for an athlete. This could mean that a person could end up with a job being decided for them before they are even born. This particular scientific advance has led to some suggestions that in the future people would be able to configure with physical features. One very good example of how gene therapy has been already used to create ‘designer babies’ is a lesbian couple from the US, both of whom are deaf, deliberately chose to have a deaf child. They were successful and now have two deaf children. Many were shocked & felt that this is against ‘nature’s way’ and ‘gods will’.
Religious groups feel very strongly about genetically modified animals (transgenic), they want nature to run without human interference and also the feeling that we should not damage with what God has designed, humans are not the creator. Both the Qur’an and the Bible teach that Humans are created by God to act as stewards over the entire world. This would include treating animals, plants and other organisms with respect as they are part of god’s creation. However in saying this others may say as humans are here to steward over God’s creation, we should be entitled to try to improve the creation by removing disorders and diseases before they take effect. Some may also say we should be able to do what we want with genetic engineering, as we are the dominant species made in God’s image.
The situation and opinions are very much similar for foods that have been modified through gene therapy. Growing populations mean there are more mouths to feed. There are currently hundreds of thousands of people starving especially in less economically developed countries. Scientists and farmers have agreed that genetically modified crops can produce more food in less space and can contain added nutrients. Food that is more nutritious can end much human suffering. Therefore some feel genetically modified foods are beneficial and should be used. One problem with such foods is although they may have been introduced to reduce world hunger, they are often infertile. This means that the farmers in the poorer countries have to buy more crops each year. Religious believers feel that nature is not ours to modify. To take genetically modified plants to the next level is progression of human techniques; however some people see this human interference as threatening biodiversity. God created organisms to reproduce sexually or asexually, not to be cloned or engineered by humans.
So What Are The Benefits and Drawbacks Of Gene Therapy?
Long Term Effects Of Genetic Engineering?
Many previous new technologies have been proved to have adverse effects unexpected by their developers, despite continuous research into the topic area. There have been numerous rumours including, that all genetically engineered foods are toxic or that all released engineered organisms are likely to proliferate in the environment. However scientists know nothing of this. According to scientists this is where the problem lies. They claim that they know of no generic harms associated with genetic engineered products. The risks of genetically engineered organisms must be assessed very carefully with huge consideration and these risks can differ greatly from one gene organism combination to another. This could explain why there is an inadequate amount of genetically engineered products available to purchase on supermarket shelves as there is currently poor development into the risks of genetic engineering.
There could be many harms and implications associated with the genetic engineering technique. First of all, the main potential effect that has to be evaluated is the potential long term adverse effects of genetically engineered organisms may have on human health. There is a growing fear that genetically engineered (transgenic) crops could bring new allergens into foods that sensitive individuals may not be able to identify and avoid. For example if the gene for one of the many allergenic proteins found in milk were transferred into vegetables like carrots, mothers who know to avoid giving their sensitive children milk would not know to avoid giving them transgenic carrots containing milk proteins, which would be a major problem and one that needs to be tackled. Another potential long term risk of genetic engineering on human health is that there are risks of antibiotic resistance. Recent studies have proven that most genetically engineered plant foods carry fully functioning antibiotic-resistance genes. The presence of antibiotic-resistance genes in foods could have serious and harmful effects. The main effect being eating these foods; if eaten they could reduce the effectiveness of antibiotics to fight disease when these antibiotics are taken with meals which effectively means the antibiotic will have no effect on the illness.
Another effect is the production of new toxins. Many genetic engineered or modified organisms have the ability to produce toxic substances. Addition of new genetic material through genetic engineering could increase the levels of toxic substances within the plants. On top of this, the addition of these new genes being added to crops can also remove heavy metals such as mercury from the soil and concentrate them in the plant tissue. Such products could pose risks of contaminating foods with high levels of toxic metals. There are also environmental risks associated with the disposal of the metal contaminated parts of plants after harvesting. It is also worth mentioning that although the addition of new genetic material through genetic engineering could pose potential effects, the removal of genes could also be a problem. To elaborate there are many positives that come with the function of genes. For instance genetic engineering might be used to produce decaffeinated coffee beans by removing the gene. However caffeine helps protect coffee beans against fungi. As with any new technology introduced the full set of risks have to be assessed with care as there could be very serious effects including the concerns over triggering cancer. However scientists are still progressing in their research of genetic engineering.
As mentioned previously there are also potential long term environmental effects of genetic engineering. For example, natural grown crops with no interference would not necessarily stay in their agricultural fields. If altered crop fields are nearby the new gene can easily move via pollen into the natural crop field meaning the gene and its traits can feed on unwanted areas. There is also the threat of poisoning wildlife. The addition of foreign genes to plants could have serious implications for wildlife in a number of circumstances. For example, engineering crop plants, such as tobacco or rice, to produce pharmaceuticals could endanger mice who consume crop debris left in the fields after harvesting. One very common application of genetic engineering is the production of virus-tolerant crops. Plants that produce viral features on their own are resistant to infection by genetic engineering, however they pose the risk of creating new or worse viruses through recombination. Similar to human health risks, it is unlikely that all potential harms to the environment have been identified however there is continuous research into the subject.
In terms of economical effects, genetic engineering has been hailed as a truly great technology. There have been claims made that it will end world hunger; and that by producing in bulk and marketing larger vegetables, the cost of purchasing food will fall. In addition there could also be the risk of trade collapse. Trade between countries could be hanging in the balance. By accepting engineered goods as a sustainable food source, there would no need for trade, as each country would be capable of producing its own. There is also a strong claim is that this technology will benefit farmers in developing countries by allowing those farmers to be more productive and grow a higher yield of crops. Many studies do agree that Genetic technologies do give farmers a higher yield, but even this is a debated issue. In relation to treating genetic disorders, the retail prices of these engineered products will be extremely high, and with nothing like it before, we could soon see genetically engineered medicines being promoted and sold globally. It is clear that genetic engineering will have a huge impact on the economy.
Conclusion
There have been truly great discoveries and advances in medical science in recent years and gene therapy and genetic engineering certainly comes into this category. The fact that the gene therapy and genetic engineering techniques consists of methods that do not require anaesthesia or even surgery and could cure genetic disorders, changing a person’s whole life is truly remarkable. Genetic technologies have given hope for untreated patients for previously untreatable diseases, and can also prevent certain diseases from taking effect in the future through the correction processes (viral vectors and Engineering). The possibilities that genetic technologies could have are never ending. Once scientist have the power to control the instructions, given to a cell, are mastered it is widely thought that any illness or disorder can be treated. For example as mentioned before, insulin can be created and grown in large quantities by using genetic engineering to manipulate method of growing a certain bacteria, thus treating diabetes patients.
Many people suffer from genetic diseases ranging from thousands of types of cancers, to blood, liver, and lung disorders, affect their standard of living and quality of life dearly. Being unable to live life like others and having to visit their Gp’s and local hospitals every week I just cannot imagine. Many hope that that this technology soon becomes a great to allow all of these innocent patients to be treated by genetic engineering, specifically, gene therapy. Genetic engineering has also opened the door for modifying crops. Higher crop yields, more nutritious food, crops grown in harsh environments, undesirable characteristics being removed and the food having a better flavor and a longer shelf life have made proponents state that that genetic engineering could sole the world hunger and starvation especially in the less economically developed countries. As demonstrated in this full report, gene therapy and genetic engineering have many potential possibilities. However as with any new technology come the drawbacks.
The prospect of altering genes brings forward questions about "playing God." The moral and ethical issue here is the human interference with the whole human genome. Although there is nothing against genetic altercation in the Qur’an or Bible, all religious people are taught that humans are created by God to act as stewards over the entire world. This would include treating animals, plants and other organisms with respect as they are part of god’s creation. By performing genetic technologies we will be clearly going against gods orders, thus triggering anger in religious people. If the gene therapy and genetic engineering was given the go ahead I predict that will be witnessing a huge debate and protest in the future.
Next is cost evaluation. Our company has unfortunate been unsuccessful in our previous attempts of treatments and medical products. This has meant the company’s profit margin has seriously been hit and is at an all time low. We are on the verge of liquidation and the profit and loss accounts of our company have proven this. However, could gene therapy and genetically engineered products be the solution and savior for our company? I mentioned previously in this report that the costs that would have to be covered if this technology was to be employed are massive. However I also mentioned that these engineered and modified products have the potential to bring in millions and be a huge success. It really all depends on close research into gene altercation, as it could also prove to be disastrous. It is basically make or break.
However, gene therapy and genetic engineering is clearly under development and is still clearly a work in progress. After all that has been said, the negatives of genetic engineering have clearly outweighed the positives, which is not a good sign. After much analysis of the techniques used to perform gene therapy and engineering , many people have come to realize that though genetic technologies helps save lives, it still has the potential to go wrong from time to time when the therapies limitations are revealed. The techniques of new genetic technologies still face difficulties. Not only does gene therapy face only difficulties, but considering ethical evaluation and close analysis of gene therapy and its costs, people have come to a conclusion that there is a likelihood of gene therapy causing harm as explained above.
This has led me to the conclude that in the future, gene therapy and genetic engineering will be a huge success and it is a truly great breakthrough in recent history for treatment of genetic disorders and will benefit many people. However at present time, the whole topic of gene therapy and genetic engineering is blurred and there is so little knowledge on the subject. For me personally it would seem wise and I recommend that the idea should be kept in thoughts but until further research and development into the implications and effects of gene therapy and genetic engineering, we should keep genetic engineered products away from the public, purely for their protection.
Glossary
