Aspects on How Gene Therapy Works - Research Paper Example

Gene Therapy Introduction Gene therapy is the therapeutic use of genes as medication. Gene therapy involves the transfer of a working copy or therapeutic genes into specific cells of a person with an aim of repairing a gen copy that is faulty (Fackelmann 239). It is important to note that gene therapy is still an experimental approach to therapy. Therefore, a lot of further research is required before the full potential of this form of therapy can be realized (Bryant, Duker and Reichel 89). The history of gene therapy and the related concepts can be traced to the 1960s and early in the 1970s. During this period, there was the discovery of cell lines which were genetically marked. Additionally, the cell transformation and how it occurred was clarified during this period as well. Developments in gene therapy were motivated by the later arrival of the recombinant DNA techniques. Clone genes were also developed which were valuable in the demonstration of how foreign genes can be used to male corrections to various defects in genes. This also revealed the importance of DNA in correcting the disease phenotypes which emanated from defective cells. Gene transfer methods and efficient retroviral vectors have shown that in vivo and in vitro phenotype correction is an effective gene therapy approaches (Dolly and Hunt 28). This is the reason why gene therapy has been justified and accepted to be applicable in clinical genetic studies which involve human subjects. Gene therapy techniques are likely to have positive impact in future scientific discoveries. This is because of its postulated future use in the replacement of faulty genes and the introduction of new genes for treatment or modification of the clinical direction of a specific medical condition. Technical Aspects on how Gene Therapy Works Genetically altered material is delivered via three major methods. The first method involves retrotransposons or retroviruses (Murray 15). Retroviruses are commonly used due to their ability to transfer their own DNA or genetic information. Additionally retroviruses are able to alter the human gene genetically. Cell DNA has parts which are able to make copies of themselves into other sections within the cell genome (Rudolph and Aslam 44). These parts which are exemplified by the yeast transposon can be used in gene therapy. However the application of transposons in alteration of genes is still under research (Judson 40). Secondly, gene therapy is made possible through blasting DNA or genes by the use of pressurized guns which are filled with helium (Bryant, Duker and Reichel 90). The helium gun is used to fire gold bullets of very small size that are coated with genes which are genetically altered (Garber 60). This technique was performed on rats with tumors in gene therapy experiments. The results revealed that the tumor cells which successfully received the inoculated altered cells demonstrated activation of coding for cytokines which played a role of activation immune cells. The third aspect of gene therapy includes liposomes (Hogan 10). This method is still being investigated where hollow molecules of fat within a solution are used in gene therapy. This methodology is used in attempts to genetically treat cystic fibrosis (Fackelmann 239). Cystic fibrosis is a respiratory tract disease which results from the buildup of chloride ions. In the gene therapy for this disease, liposomes which are coated with genes that are genetically altered are inhaled. The genetically altered genes are designed to prevent chloride ion buildup. The experiments of gene therapy have revealed that the ion levels within the respiratory tract decreased significantly with the inoculation of the genetically modified genes. The advantage of this method is that it poses less potential risk as compared to retroviruses. Positive Aspects of Gene therapy Gene therapy is advantageous because it can be used to treat individuals who have congenital genetic disease. This can be achieved through the replacement of the non-functional gene as inherited from the parents by a functional gene by the use of gene therapy (Rudolph and Aslam 45). It is in this regard that it has been argued that gene therapy is important in enabling people with genetic defects to live normal lives and contribute positively to the society. More significantly, gene therapy is a means of eradication of genetic diseases from the future generations (Dolly and Hunt 28). The remedy that gene therapy has for genetic disorders means that when non functional genes are replaced by functional ones, the transfer of the gens to the subsequent offspring is prevented. Therefore it is argued that gene transfer is significant in promoting advantageous genes within the genetic pool of a generation (Murray 15). Genetic therapy has been used in the management of cancer. It is through the techniques of gene therapy that cancer patients can benefit from the insertion of various genetically altered genes or vectors into their genome. This techniques are being investigated with a view of coming up with a permanent cure for cancer. Therefore gene therapy would be the most significant approach of therapy which will be used to come up with solutions for the suffering that is experienced by cancer patients. The use of gene therapy in the treatment of cancer is exemplified by the gene therapy of unborn babies whose mother has breast cancer or who have strong cancer history within the family. It has been argued that gene therapy among infants has been used to significantly reduce the chances of development of cancer later in life (Thomas and Klibanov 27). Unless the cancer predisposed children are exposed to numerous carcinogens, it has been said that the possibility of developing cancer is reduced significantly (Garber 63). Gene therapy is likely to replace surgery or the use of drugs in the treatment of various diseases (Thomas and Klibanov 30). The experiments in gene therapy are focused at allowing doctors to insert genetically modified genes into the cells of the patient. In this sense, treatments will be achieved without the painstaking and risky surgeries. Moreover the side effects which characterize some drugs would be avoided through the application of gene therapy in treatment (Bryant, Duker and Reichel 91). Gene therapy targets the treatment of diseases which result from the mutation of genes (Garber 58). Therefore doctors will be able to fight disease by the introduction of new genes into the genome of the patient so that the mutated gene can be replaced with normal ones. The promise that gene therapy has as a solution for various diseases and disorders, it is notable that the technique is still risky with various cons as discussed in the following section. Negative Aspects of Gene therapy The viral vectors which are used in gene therapy such as retroviruses pose threats to human life which include possible inflammatory and immune responses and toxicity (Hogan 10). Most gene therapy scientific investigations and studies use viral vectors as the carriers of choice for the genetically modified genes. This reveals a negative aspect of gene therapy where patients are predisposed to health risks. It has been argued for example that viral vectors of may resume their ability to cause disease once they have been inoculated to the patient during gene therapy (Rudolph and Aslam 44). These arguments justify the opposition which has been staged against gene therapy by its opponents. The benefits of gene therapy are short term (Murray 17). This is because the functionality of the genetically modified DNA which is introduced to defective genes must exist for a long time for the therapy to be effective. Additionally the target cells must maintain their stability and functionality until the therapy is successful in alteration of the defective genes (Dolly and Hunt 28). However, the nature of human cells is rapid mitosis or division. This therefore illustrates that gene therapy has limited guarantee of longevity of its treatment potential. Furthermore, the rapid division or multiplication of cells requires a patient to undergo multiple DNA inoculations during gene therapy. In light of these demonstrations, it is said that the benefits of gene therapy are short term (Fackelmann 239). Cases of integrating DNA into a wrong focus within human genome have been reported during gene therapy experiments (Judson 40). It is in this regard that it is evident that gene therapy has a possibility of inserting mutagenesis and hence leading to induction of cancer (Rudolph and Aslam 45). For example if genetically modified genes are replicated into tumor suppressor cells or genes by mistake during gene therapy, the patient is likely to develop cancer. It is therefore the risk of gene therapy mediated tumors that these procedures have been opposed as risky to the well being of human beings. Nonetheless, it is argued that such a problem would be overcome by addition of tumor suppressor DNA into the patient cells. Challenges and Unknown Aspects in Gene Therapy The challenge which the successful treatment through gene therapy demonstrates is due to the fact that the condition of the patient must be understood very well (Hogan 10). Additionally, it is necessary for the identification of the underlying faulty gene for the condition which a patient is suffering from. Scientists have not been able to locate the underlying gene for every disease which makes it challenging to achieve successful gene therapy for some disorders (Bryant, Duker and Reichel 92). Furthermore, a working copy of the gene responsible for a particular disorder must be known and made available for gene therapy to be successful. Another challenge that the treatment in gene therapy faces is the identification of the cells which requires treatment and accessing or delivering vectors during gene therapy procedures (Dolly and Hunt 28). It is the somatic or body cells which are normally targeted during gene therapy (Fackelmann 239). Sperm or egg cells have not been successfully targeted by gene therapy. This illustrates that scientists have not achieved success in adequately preventing some disorders from being passed to future generations. Because somatic cells and their treatment have no impact on subsequent generations, it is arguable that scientific research has gaps in the target of egg and sperm cells in gene therapy. This challenge is related to the fact that scientists have not known how they would alter sperm or egg cells without causing more negative implications on the next generation than the desired effects. It is in this regard that the investigation of gene therapy which targets egg and sperm cells and their manipulation has received immense debates on the ethical considerations which are related to the alteration of sperm or egg cells. Gene Therapy and Future Treatments There is a lot of potential for gene therapy in the treatment of various diseases and inherited conditions. For example conditions such as cancer and HIV/AIDS which have proved difficult to obtain conventional treatment reveal the potential that gene therapy has for their successful treatment (Murray 18). Moreover the approval for gene therapy studies for the treatment of diabetes and obesity would lead to a significant potential of managing these conditions successfully. An illustration of gene therapy in HIV/AIDS is demonstrated by a Germany patient into whom HIV resistant CCR5-deficisnt cells were introduced during a stem cell transplant. This treatment was successful because the doctors have not been able to detect that virus within the patient anymore. It is in this regard that it can be pointed out that diseases which cannot be treated by conventional pharmacological interventions would be managed successfully through gene therapy. It has further been revealed that the future f human care is likely to depend on gene therapy especially in the treatment of viral infections. This is attributed to the power within gene therapy where it can be used to alter the human genetics makeup rather than directly attacking the viral causative agents. Conclusion The therapeutic use of genes in treatment of various diseases has developed historically with the arrival of the recombinant DNA techniques. Through gene therapy, DNA is used in correcting the disease phenotypes which emanate from defective cells. Genetically altered material is delivered via three major methods. These are the use of retrotransposons or retroviruses, blasting DNA or genes by the use of pressurized guns which are filled with helium and the use of liposomes. The positive aspects of gene therapy include its use to treat individuals who have congenital genetic disease and hence eradication of genetic diseases from the future generations. The potential application of gene therapy in the treatment of cancer, HIV/AIDS, diabetes and obesity are some of the indications of the benefits which studies on this technique have proven to have in health care. Nonetheless, gene therapy has negative aspects such as the risks of viral vectors to cause disease within the patient and ethical concerns which are related to the alteration of egg or sperm cells. Finally, scientists are faced with various challenges in gene therapy processes such as the unknown causative genes for some diseases which make the application of gene therapy unable to achieve the desired therapeutic effect in some cases. Works Cited Bryant, Juanita, Jay Duker and Elias Reichel. "Gene Therapy for Retinal Disease." Review of Ophthalmology 19.4 (2012): 89-92. Print. Dolly, Aggarwal Parasrampuria and Connie Hunt. "Therapeutic Delivery Issues in Gene Therapy, Part 1: Vectors." Pharmaceutical Technology 22.4 (1998): 28. Print. Fackelmann, Kathleen. "Gene Therapy for Breast, Ovarian Cancer." Science News Apr 11 1998: 239. Print. Garber, Ken. "High Stakes for Gene Therapy." Technology review 2000: 58-64. Print. Hogan, Jenny. "Can Gene Therapy Beat HIV when Drugs Don’t Work?" New Scientist 2004: 10. Print. Judson, Horace F. "The Glimmering Promise of Gene Therapy." Technology review 2006: 40. Print. Murray, Bruce. "Building Better People: The Truths and Myths of Gene Therapy." The Quill 89.5 (2001): 15-18. Print. Rudolph, Benjamin H. and Viquar Aslam. "Gene Therapy: Are You a Believer?" Biopharm International 14.8 (2001): 44-45. Print. Thomas, M. and A. M. Klibanov. "Non-Viral Gene Therapy: Polycation-Mediated DNA Delivery." Applied Microbiology and Biotechnology 62.1 (2003): 27-34. Print. Read More