Gene Therapy as Effective Medical Intervention - Coursework Example

GENE THERAPY Introduction Biotechnology has its roots in our history. Human mankind always is in the search of enhancing his standards of living based on scientific principles. Biotechnology is one of the sophisticated techniques to achieve healthy future. It has impacted on all the kingdoms from microorganism to humans. Genetic engineering (GE) gives us focused path in designing new varieties of microbes, plants, animals and human being by understanding their genetic make up. Genetic research has revolutionized the world and becoming the key solution for combating genetic disorders. Genetic disorders are characterised by presence of simple abnormal genes giving rise to genetic diseases like Huntington’s disease and cystic fibrosis (CF); and more complicated factors involved along with defective gene leading to complicated health issues like cancer and diabetes mellitus (Sade and Khushf, 1998, p. 406). Gene therapy is becoming burgeoning field in biotechnology which is offering unparalleled solutions to gene related disorders at the molecular level. Gene therapy refers to the therapeutic replacement or rebuild by repairing abnormal genes in the human cells (Stafford and Mannor). In addition to the monogenetic diseases (disorders due to one gene) that could be positively treated by gene therapy, it could also offer potential remedies for more complex diseases in near future. The first approval for gene therapy protocol was given in 1990. The diseases which are under investigation are cancers, HIV infection, cystic fibrosis (CF), Gaucher disease, severe combined immune deficiency (SCID) due to adenine deaminase (ADA) deficiency, familial hypercholesterolemia, rheumatoid arthritis, etc. In the future, gene therapy could be possibly exploited for other diseases like muscular dystrophies, particularly Duchenne, and hemoglobin diseases like sickle cell anemia and betathalassemia (Sade and Khushf, 1998, p. 406). The techniques used in gene therapy for gene manipulation are broadly categorized into three types. Gene addition or gene insertion is the technique in which normal version of an abnormal gene is inserted into the genome of the targeted cells, without focusing for its insertion in chromosomes. This is the only widely used method for human genetic manipulation. When newly inserted gene is expressed, it has ability to produce enough quantities of a missing or defective protein or enzyme, thus "curing" specific gene associated disease. This random technique might lead to disruption of the function of another vital sequence. Therefore, to avoid this potential danger other two methods are also being explored (Stafford & Mannor). Gene repair technique involves the replacement of abnormal parts of DNA in problematic genes in their regular chromosomal site. Thus, the abnormal DNA sequence is chemically altered to acheive normal duplicate gene sequence in the cell. This method might prohibit potential danger of disrupting the functions of other genes due to introduction of tailor-made normal DNA sequence (Stafford & Mannor). The technique which is not in use in human experiment is the gene replacement or gene surgery technique. This technique eliminates defective genetic sequence precisely by excision. This part is replaced by a normally functioning cloned copy of the sequence (Stafford & Mannor). This sophisticated technique might be the ideal approach for gene therapy because of least side effects and is vital from the point of the eventual deployment of germ-line therapy (Sade & Khushf, 1998, p. 407). The specific genetic manipulation in somatic cell therapies are based on the nature of a particular disease and other interlinked attributes. Target cells are lung, liver, white blood, endothelial, and cancer cells (Sade & Khushf, 1998, p. 406). In the germ-line technique, the genes of germ cells are altered to pass on positive clinical outcome in the subject’s future generations. The alteration in the target cells in the somatic therapy could be done in vitro (66% methods are based on in vitro procedures) or in vivo (33% methods are based on in vivo procedures). Retroviral vectors (RNA viruses) are attached to new genetic material to introduce into target cells. Introduced viral RNA in the host’s genome is replaced by human RNA that transcribes cDNA containing the desired human gene. This functional cDNA is passed on to daughter cells at cell division. Other vectors which can be used are adenovirus and adeno-associated virus, and nonviral agents, like liposomes that encapsulate human DNA, and naked DNA (no protein envelope) are introduced directly into cells by microinjection (Sade & Khushf, 1998, p. 406). Pros and Cons of gene therapy Advantages The innovative approach of gene therapy has given immense promise in terms of eliminating virtually all gene linked diseases. Somatic gene therapy is a specialized personalized medical intervention. The frequent administration of expensive ADA is the present medical treatment available to treat SCID. Addition of the ADA gene in such patients might able to produce ADA inside the body itself by spending money onetime on such therapy. CF patients could achieve positive clinical outcome by genetically altering the patient’s self lung cells to prevent damage caused by CF genes and exhibit reversal of the CF pathophysiology (Sade & Khushf, 1998, p. 407). Thus, the patient has normally functioning pulmonary cells by avoiding expensive lung transplant procedure which has inherent immunosuppression to prevent rejection (Sade & Khushf, 1998, p. 407). Until now, germ-line gene therapy is the only effective treatment which can be thought of for treating certain genetic problems including tumors like in retinoblastoma. Each person should that are capable of forming tumor should be reached and treated. The gene defect in gametes, zygotes, or multi-cell pre-embryos should be treated to make each cell normal. Removal of tumor genes might prevent future such genetic problems (Sade & Khushf, 1998, p. 409). Somatic cell gene therapy has definite positive prospects for treating diseases like cystic fibrosis or diabetes. But, if germ-line gene therapy could be potentially used, then it might avoid future generations to suffer form the same problems of current generations due to removal of root cause of the problem i.e. deleting harmful genes. Thus, germ-line therapy has promising future to produce healthy future generations by adopting disease prevention strategy (Sade & Khushf, 1998, p. 410). Germ-line therapies have ability to affect human evolution by extending lives to a greater degree and in a shorter time frame than normal medical treatments than other interventions (Sade & Khushf, 1998, p. 409). It is possible to create population which is free of traits such as nearsightedness, shortness, or athletic and academic inability. Each parent can deliver specialized dream child by selecting choose their childs gender, physical appearance, athletic ability, and academic proficiency (Stafford & Mannor). Risks An unknown new way and rudimentary knowledge give rise to whole set of new questions. These problems might be such that we would have never thought of and we are not able to see in advance all the pitfalls that will almost inevitably take place. The consequences of the genetic engineering methods and gene therapy entail unprecedented risks to human health and the social values. Gene therapy is facing criticism due to its inherent powers to harm life for all future generations. Somatic gene therapy research should focus on possible adverse effects and also the long term effects on the body as a whole (Bruce). The secondary effects of introducing normal gene by manipulating or replacing defective gene should be cautiously studied. Though such strategy recovers and corrects abnormal function by as stimulating a vital protein potential secondary effects should be assessed and monitored over a due period of time (Bruce). The current tool might cause infection by viral vectors. The modifications might hamper other vital gene functions causing other major health problems like cancer (Stafford & Mannor). Well studied evidences in terms of such effects and positive reasonable clinical outcomes would boost this novel treatment. In addition to safety and efficacy, other ethical issues which are considered in somatic gene therapy research are like other clinical settings which includes: weighing potential harms and benefits, establishment of procedural fairness in selection of patients for research, assurance that consent to experimental treatments is informed and voluntary, and protection of privacy and confidentiality of medical information (Sade & Khushf, 1998, p. 407). Gem-line therapy has diverse ethical issues like it could lead to imbalance in the sex ratio among population and disruption in genetic natural variation. An engineered progeny with desirable attributes by introducing desirable genes might cause inbreeding problem. These factors could result in enormous genetic diseases. It should be kept in mind that by eliminating certain "undesirable" traits (such as sickle-cell trait), abilities and possibilities of vital adaptations to certain environments could be hampered. For example, getting rid of sickle cell trait might elevate malaria cases by interfering with mechanisms related to natural immunity to the disease (Stafford & Mannor). Biotechnology industry is always suffering from commercial and national governments pressures. To patent the mapped sections of the human genome is live example of unforeseen future disruption (Bruce). The patenting and commercialization of gene therapy might create adverse socioeconomic impact. Conclusions Undoubtedly gene therapy is the most potent effective medical intervention, but due to the hazards entailed by gene therapy, it should be used cautiously. The person should be scrupulously studied for both the advantages and the disadvantages of gene therapy. Somatic cell gene therapy research might not pose the potential risks and therefore is ethically acceptable. Its efficacy should be used for the patients suffering form a genetic disease. Germ-cell therapy should be well researched for future side effects before experimenting on humans. Use of Germ-line gene therapy should be restricted to treat genetic disorders and not human attributes. This would help to avoid gender imbalances, inbreeding problems, reduction in genetic diversity, and elimination of adaptations associated with carrier status of a disease (Stafford & Mannor). It is necessary to identify legitimate issues and potential threats of gene therapy, and they should be openly discussed with the public along with the positive effects of gene therapy in the interest of public and environment. The selecting gene therapy treatment should be optional for an individual. The genetic research based on gene therapy should be regulated globally so that basic ethical norms are not violated and potential risks are minimized. Biotechnology related individuals which include; scientists, government and consumer society should possess a sense of awareness and responsibility towards human mankind. The use gene therapy should be done constructively and make the world heaven for everybody by avoiding mishap and controversies. References Sade RM, Khushf G. Gene therapy: ethical and social issues. J So Carolina Med Assoc 1998;94(9):406-410 Kate Stafford and Michael Mannor. Gene Therapy. http://library.thinkquest.org/17109/therapy.htm Bruce, Donald M. Moral and Ethical Issues in Gene Therapy. Society, Religion and Technology Project. http://www.srtp.org.uk/genthpy1.htm. Read More