Over the years genetic disorders and gene-related illness have been responsible for high mortality rates and reduced quality of life. Some of the congenital abnormalities manifest quite early, and there are minimal hopes for survival in these children, this causes much pain to their families because management option is limited and there is very little at their disposal to modify such conditions. Scientists are developing a relatively new technique that will give hope to the hopeless and make life better. Genetic disorders can be due to misalignment, missing genes or excess of a gene. Genetic therapy works at the elementary level of heredity to replace the defective genes with new ones.
Gene therapy is the insertion of new functional genes into an individuals cell or tissue to replace the defective one and modify a hereditary disease. This is a new research area, and much research projects are still in the infancy or trial stages; however, expectations are high, and we would potentially do away with genetic diseases soon enough. Developed in 1972, gene therapy can be of two type; somatic gene therapy and germline gene therapy. It is especially promising in treating genetic disorders such as muscular dystrophy and cystic fibrosis. Critics have their say and look at the two sides of the story will lead us to weigh risk: benefit ration. Gene therapy pros and cons can be quite decisive on whether or not we should embrace gene therapy.
Research into gene therapy has been ongoing for decades now, and there is light at the end of the tunnel as gene therapy is associated with the following advantages;
Genetic defect occur even after thorough screening and many people have their lives compromised or limited by such disorders. In the United States alone as at 2009, 3% congenital disabilities were recorded in all births, the joy of having a child is suddenly lost and replaced with the agony and struggle of maintaining such demanding life. Individual patient and family are pessimistic as efforts to change the condition by traversing through different levels of healthcare are always thwarted since no cure exists for such situations. Gene therapy intent to correct such birth defects responsible for more than half of infant mortality is laudable. We can be confident that all the unborn babies will be delivered safely and grow to their prime. Besides, gene therapy promises a cure for the chronic illnesses that are currently incurable for example cancer that causes agonizing pain in many.
By replacing a defective gene with a functional gene in a disease like cystic fibrosis, there are limited chances of remission, and this is usually a one-off treatment that will see you symptom-free for life. Furthermore, gene therapy is not just a remedy to the individual suffering from a given condition, but it covers the entire generation. When you remove a gene predisposing one to breast cancer they wont transfer the defective genes to their offspring but the new functional gene.
Some of the initially incurable diseases can be managed and possibly eliminated when we involve gene therapy. Alterations to gene especially the reproductive genes using the germline method can help in avoiding transmission of defective genes and thus no further incidences of disease. Parkinsons disease, Huntingtons disease, and Alzheimers disease are but a few conditions that are likely to be eliminated by gene therapy.
Not only does gene therapy focus on diseases. Conditions that may make life unlimited such as infertility are also accounted for, and it is projected that soon enough gene therapy should activate reproductive genes and allow you to have children.
Gene therapy promises much potential in the medical field ranging from relieving the pill burden to modifying phenotypes in cosmetology.
Although gene therapy has a potential for treating several ailments and improving life, this is a relatively new technique and involve several safety concerns thus it should be carefully embraced. Some of the notable disadvantages of gene therapy include;
As at now, gene therapy is at the developmental stages, and most experiments are done on the animals with the hope that success will be reflected in humans. However, anything can happen, and gene therapy can fail to work thus limit your capabilities or even worsen the condition. Incompatibility issues and immune response can also lead to failure of the procedure. The massive ongoing research will leverage the concerns and provide a technological-based method with a high certainty of results.
The cost of performing gene therapy is likely to be high because it involves sophisticated equipment and high-level expertise. Such a fee may not be affordable to many, and this will create socioeconomic segregation as the rich will be disease-free while the poor remain to bear the burden of such illness. A solution to this would possibly be extensively integrating technology in the process since technologically based therapy may prove more cost-effective even when compared to the other therapeutic options.
Superb as it may look, gene therapy may be short-lived. Nature is unpredictable, and sometimes it may take its course for the worse. Just like the antibiotics were novel at the time of invention, it is possible that the use of gene therapy may gain resistance as well. There is no guarantee that gene therapy will fulfill its expectations to treat explicit disorders, performing gene adjustments could create new defects for future generations without realizing it; thus it can be a double-edged sword.
One of the greatest hindrance to the advancement of gene therapy is the opposition on the basis that it can open room for unethical science which may be demeaning to the human race. The thoughts that gene therapy creates a perfected human has been very controversial.
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