Genetic Therapy and Engineering - Term Paper Example

Student’s Name: Tutor: Course: Date: Genetic Therapy Introduction Gene therapy is the process by which genes are implanted into the cells of a person to treat various types of diseases. This therapy is used by doctors instead of conventional medicine or surgery to treat different types of diseases in humans and animals. During gene therapy, doctors use various processes to inactivate dysfunctional gene which is replaced with a healthier gene. This process counters the destructive effect of the mutated gene and ensures that disease causing organisms in the body are destroyed. As a result, a patient is able to enjoy a healthy lifestyle free of different types of diseases. Gene therapy is mainly used to treat diseases caused by gene defects in the body that are harmful to human health. This therapy is used to treat hemophilia, sickle cell anemia, cancer and different types of fibrosis. The process is complex and requires large sections of DNA which are modified and then introduced to a dysfunctional human cell. History The earliest form of gene therapy occurred in September 1990 in the US. It was done on a little girl who had a genetic defect which affected her health. After the process, her immune system became weaker but she was able to overcome these deficiencies later. Afterwards, doctors uncovered new procedures of treating blood disorders, cysts and cancer. New apparatus for carrying and implanting DNA in human cells were developed which were able to insert tiny DNA particles into human nuclear membranes (Kilner, Pentz and Young 67). Another experiment proved that the therapy was able to treat sickle cell anemia in mice. Scientific researchers were able to discover that gene therapy was able to induce the body to produce fetal hemoglobin which made it possible for humans to counter the negative effects of sickle cell anemia. In 1992, the first procedure of gene therapy that relied on hematopoietic stem cells was done in Milan, Italy. These cells were used to treat hereditary diseases in patients and they later had a negative impact on their immunity. This process helped medical researchers to uncover new methods of dealing with severe combined immunodeficiency. In 1993, stem cells mixed with retroviruses were used to treat an infant who was suffering from SCID in the US. Ten years later, scientists discovered that genes could be implanted into a person’s brain to treat Parkinson’s disease (Kilner, Pentz and Young 72). In 2006, scientists were also able to treat melanoma in patients by inserting T cell genes which were able to inactivate cancer cells. More discoveries by doctors and medical practitioners showed that gene therapy was able to treat defects occurring in myeloid cells in humans. The development of immunotherapy that relied on genes for the treatment of HIV was also developed in 2006. The process revealed that it was able to increase the viral load in patients who had undergone the process and it was approved for further tests by the FDA in the U.S. In 2007, gene therapy for hereditary retinal disorders was done in London. It revealed that the use of this therapy was beneficial to patients because it helped them increase their vision and had no side effects on their long term visual ability. Further clinical studies in 2008 and 2009 revealed that gene therapy was an effective method of treating visual problems in humans and animals. A transplant done in a patient in 2010 helped to boost his hemoglobin levels and this reduced his overdependence on transfusion to survive (Rosenberg and Rosenberg 53). A patient was also treated and cured of HIV by hematopoietic stem cell transplantations. However, other doctors argued that this process had negative side effects because it weakened the bone marrow. The process was also used to treat lymphatic leukemia in patients in Philadelphia in 2011. T cells were inserted into the patient’s body to counter the harmful effects of genetic disorders. More studies have also been conducted to find out if gene therapy can be used to treat coronary artery disease and the resulting damage that this disease does to the human heart (Rosenberg and Rosenberg 58). Gene therapy was also approved by the FDA to treat thalassemia. Studies are being conducted to find out how this mode of treatment can be used to treat other diseases. Recently, several patients have been treated using gene therapy and they have managed to overcome different health problems they are facing. Definition and Diseases Treated Using Gene Therapy Gene therapy is the insertion of genes as an agent of treatment into a person’s cells to treat defects caused by individual cell dysfunction. The most commonly used genes are in form of DNA cells which act as vectors that correct different types of mutations after they are inserted into a person’s body. This therapy is used to treat different types of hereditary and acquired diseases in the human body. There have been more than 1,000 clinical trials which have been carried out to find out the benefits of using gene therapy to treat different types of diseases in the body (Rosenberg and Rosenberg 61). The major three diseases which can be treated using gene therapy are: Gene therapy can be used to treat cystic fibrosis. This is a hereditary disorder that is passed on to children by one of the parents who is a carrier of the defective gene that causes it. Patients that have this disorder suffer from coughing, wheezing, respiratory difficulties and loss of weight. Doctors diagnose these patients by assessing the amount of salt in their sweat to find out if they are suffering from this disorder. People with this disorder also have large amounts of trypsinogen proteins in their blood and doctors use this as an indicator to show whether a person has cystic fibrosis or not. The genetic therapy for treating this disorder involves the insertion of a cystic fibrosis regulator (CFTR) into a patient’s cells in the lungs. The healthy CFTR genes are able to correct the negative effects of the disorder by making harmful CFTR cells inactive in the body (Kelly 40). Gene therapy is also used to treat different types of cancer in the human body. Therapeutic gene vaccines and other therapies have been tested by scientists in different countries to find out if they are able to get rid of harmful cancer cells in the body. Tests have been conducted to develop gene vaccines for prostate, pancreatic, breast, ovarian and skin cancer. T cells are genetically modified and inserted into the body to counter the destructive effects of defective cells that cause melanoma in patients (Kelly 43). Technologies are still being developed to enable doctors to be able to insert these cells in a patient’s body without too many side effects. Some of the technologies which have been recommended involve the insertion of genetically altered vectors into the human body to make cancer cells inactive. New studies are also being undertaken to find out how gene therapy can be used to treat skin cancer in humans. In the future, gene therapies will be the most preferred cancer treatments by many oncologists. Gene therapies are also used to treat hemophilia. Patients who have hemophilia are not able to form blood clots and this makes them susceptible to various episodes of internal and external bleeding which are dangerous to their lives. This disease can be treated by inserting a therapeutic gene into a patient’s liver cells to enhance its ability to stimulate blood clotting functions in a patient’s body. This makes it easy for a patient to manage dangerous symptoms of the disease in his body (Budinger and Budinger 77). There are different types of gene therapies which are used to implant life saving genes into blood clotting cells that are found in the liver and other organs in the body. These therapies enable a patient to live a healthier lifestyle without getting slowed down by diseases. Moral Dilemmas There are several moral and ethical issues which are related to genetic therapy treatments. One of the most common dilemmas associated with gene therapies is that it produces inconsistent results in patients when administered. Experiments involving the insertions of ADA genes in children have produced different results in different health institutions and this has made it difficult for medical researchers to come up with a universal treatment process. Some doctors have also faced difficulties in developing similar but more effective genes to be inserted into various body cells that have different types of diseases (Budinger and Budinger 80). As a result, they are not able to choose the most effective alternative genes to neutralize the threat caused by harmful genes in the human body. Therefore, their efforts to develop effective gene therapy solutions have been frustrated by these problems. Some gene therapies affect the development of a fetus when they are inserted into a pregnant woman. As a result, the baby is likely to be born with various complications which may affect her growth negatively. Germline gene therapy makes unborn children to be subjected to gene therapies without their choice and this violates their constitutional rights. Germline gene therapy is done to egg and sperm cells to ensure a parent does not pass on a disease causing gene to his offspring before conception (President’s Council 63). The child that is born will not likely have the same genetic components as his or her parent. Germline gene therapies try to alter the course of nature and this provokes religious and moral debates about their usage in different societies. Transgenic combinations which are used in gene therapies may cause significant alterations to people’s cells. There are also other health risks which are likely to arise which may have an effect on the health and well being of the recipients of these transgenic cells. Some scientific experts have argued that these therapies may alter some physical and emotional characteristics in humans making them acquire new traits and behaviors. As a result, this situation may lead to the development of new health disorders that may be difficult to treat in the long term (President’s Council 67). Some scientists and ethical campaigners argue that this crosses a moral line because it become difficult to define various species based on their inborn characteristics. They insist that these therapies should not be used on a large scale because they are likely to cause some plant and animal species to evolve into strange organisms. Some scientists have also argued that there are risks which are likely to occur due to the experimental use of animals in gene therapy studies. They argue that this violates animal rights because they are not able to speak for themselves to defend themselves against actions by scientists. They also insist that genetic alterations on animals should not be allowed because this has a negative consequence on biodiversity on planet earth. There are long term risks which are likely to occur to the environment and this threatens the wellbeing of all animals that live on earth (Trent 43). In effect, endangered species may not be able to reproduce and multiply their numbers when they are used in such experiments. Therefore, this violates the sanctity of life because some species are likely to become extinct due to their exposure to various genetic experiments. Some scientists have also called for the banning of gene altering technologies. They argue that this may cause some people to use genetic engineering to alter the traits of their children to give them a competitive edge over normal humans. As a result, some parents may alter the genetic components of their children to make them develop their sports, business and physical combat activities (Trent 49). However, proponents of genetic technologies insist that they will ensure parents are able to impart new skills to their children at a young age and this will help to solve a myriad of problems in different societies. These bright and intelligent humans will also be more inclined to do good deeds to improve the welfare of other people living in different societies. They also insist that people should not be denied their civil liberties that improve their lifestyles in various societies they are living in. Some technologies have also been found to spread various types of diseases in humans. The transplantation of genetic cells into various organisms can alter their physical immunities thereby resulting in the development of new diseases. Some diseases which are common in animals may be transferred to humans who receive these treatments and they are likely to infect other people who come into contact with them (Trent 57). These procedures are likely to have devastating effects on different populations because they may increase the development of diseases that are difficult to eradicate. Therefore, it is important to evaluate safety implications of these technologies before they are used to deal with various health disorders in people and animals. Advantages and Disadvantages Advantages Gene therapies help people who are born with genetic diseases to have a chance to lead normal lives. They correct genetic defects in humans making them less susceptible to various hereditary diseases that have negative effects on their wellbeing. Gene therapies offer solutions to medical disorders and diseases which in the past have proved incurable. They make disease causing cells and microbes in human and animal cells inactive thereby giving them a chance to live healthier lives. Gene therapies also implant positive viral vectors into the human body. The new gene is able to insert its genome into the dysfunctional cell and this guarantees a person a longer and healthier life. Gene therapies when used effectively can reduce costs incurred by patients who have tried conventional medicine without any changes. As a result, this therapy makes a patient to get a long term solution to the health problem he is facing and this ensures that he does not make repeat visits to health facilities to seek treatment. Germline gene therapies treat a person’s genetic disorders and they also limit his ability to pass on these genes to his offspring. As a result, this reduces the occurrence of various hereditary disorders that are passed on from parents to their children. These therapies make it possible for people to protect their children from genetic defects that are likely to impede their physical and intellectual development. Germline gene therapies improve the reproductive abilities of mothers who are about to give birth. They ensure that their embryos are in a good condition to ensure they are able to carry their pregnancies to full term. Disadvantages Some genetic scientists may abuse the freedoms they enjoy to develop technologies which have negative consequences on humans. As a result, they may conduct experiments on humans without taking safety precautions into consideration thereby endangering their lives. Some patients are not in a position to give informed consent to doctors and other medical experts to try out these therapies on their bodies or those of their children. As a result, they may not be able to understand the long term health implications of using these technologies to correct various health defects. Some technologies and processes are likely to give rise to new diseases in humans and animals. Recipients of these transgenic genes which are transplanted from animals to humans are likely to acquire new viruses and bacteria whose cures have not been found. Germline gene therapies can endanger the life of an unborn baby especially when inappropriate techniques are used to repair defective embryos. As a result, a child may acquire severe health risks which may have negative effects on his growth and development. Advanced genetic engineering processes may be used to alter natural characteristics of animal and human species making them more violent and destructive. Therefore, these species may be controlled evil scientists to control other people to achieve their own selfish interests. Some genes may be transplanted in the wrong DNA genes of the human body and this is likely to have a negative effect on the health and wellbeing of humans. Therefore, this may give rise to more complications in the body of a person receiving these additional genes. Conclusion Genetic therapies have a lot of significance on advances being made in biotechnology. Therefore, they need to be well utilized to ensure they serve the interests of human and other living species on planet earth. They make it possible for medical specialists to cure different types of genetic disorders that are inherited by children from their parents. Gene therapies will change modern science in future because new discoveries will be made which will make them cheaper and easy to administer to a large number of patients. Works Cited Budinger, Thomas F., and Miriam D. Budinger. Ethics of Emerging Technologies: Scientific Facts and Moral Challenges. San Francisco: Wiley, 2006. Print. Kelly, Evelyn B. Gene Therapy. Westport, CT: Greenwood Publishing Group, 2007. Print. Kilner, John Frederic, Rebecca Davis Pentz, and Frank E. Young. Genetic Ethics: Do the Ends Justify the Genes. Cambridge: Wm. B. Eerdmans Publishing Company, 1997. Print. President’s Council on Bioethics. Human Cloning and Human Dignity: An Ethical Inquiry. Washington, DC: Government Printing Office, 2002. Print. Rosenberg, Leon E., and Diane Drobnis Rosenberg. Human Genes and Genomes: Science, Health, Society. London: Academic Press, 2012. Print. Trent, R.J. Molecular Medicine: An Introductory Text. London: Academic Press, 2005. Print. Read More