Gene Therapy

Gene Therapy
Gene therapy is the procedural technique that uses genes to prevent or treat diseases. Due to advancement in technology, this procedure is being used by physicians to cure a disease by introducing DNA into the patient’s cells rather than using surgical procedure or drugs. Gene therapy will soon enhance the correction of gene disorders, prevent cardiovascular diseases, eliminate cancerous cells and eliminate infectious pathogens. However, it is important to differentiate between gene therapy from the use of genomics to research on new drugs and diagnosis procedures, although the two are similar in some aspects (Brown & Brown, 2016). This paper is a discussion of gene therapy in details.
Gene therapy is aimed at introducing genetic material into cells as a replacement for abnormal genes. If the mutated gene contributes to problems to the necessary protein making it being missing or faulty, gene therapy is capable of introducing a similar replica of the DNA to enable the proper functioning of the protein. A gene that is injected right into a cell usually is not functional. Instead, a carrier known as a vector is genetically improved to provide the DNA. Particular pathogens are usually used as vectors as they provide the new DNA by infecting the cell structure. Therefore, the pathogens are adjusted, so that they do not contribute to diseases when injected in individuals. In relation to this, various kinds of viruses such as retroviruses, combine their genetic composition into the DNA in the human genome (Ibraheem, Elaissari & Fessy, 2014).

Moreover, other viruses including; adenoviruses, insert their genetic composition into the central part of the cell, but the DNA is not added into the cell. Then, the carrier is injected into a particular body tissue body, where it is absorbed by particular cells. Besides this, a section of the patient’s cell can be extracted and directed to the pathogen in a research facility. The cells holding the pathogen are then inserted into the patient. If the cure is a success, the new genome provided by the carrier will form a protein which is functional. It is important to note that scientists should solve numerous problems before gene therapy becomes a practical procedure for treating disorders. For instance, researchers should research on better methods to provide DNA and target them to certain cells. They should also confirm that the new DNA is entirely regulated by the body.
Safety of Gene Therapy
Gene therapy is still under investigation to establish whether it can be used to cure disorders. Recent studies are estimating the risk of gene therapy. Also, future research will examine whether it is an appropriate procedure. Numerous research also has established that this procedure can lead to serious health risks, including; cancer, toxicity, and inflammation. Since the procedures are new, some safety measures may be irregular. However, institutions, research scientists, and regulatory authorities are working to determine that the process of gene therapy is less risky for it to be experimented on humans. Detailed regulations, guidelines, and federal laws enhance protection of individuals who are involved in research studies. The U.S Food and Drug Administration (FDA) is given the mandate for controlling all the gene therapy merchandises in America and supervises studies in this area. Scientists who wish to carry out a procedure in a laboratory must first obtain clearance from the FDA (Brown & Brown, 2016). The FDA has the mandate to reject laboratory procedures that are assumed of being unsafe for members. Additionally, the National Institutes of Health (NIH) also is involved in enhancing the safety of gene therapy studies. NIH provides regulations for institutions and supervisors to follow when carrying out laboratory tests with gene therapy. These rules state that laboratory tests at institutes that receive funds from NIH for this particular study must be assigned by the NIH Office of Biotechnology Research. The procedure for every lab test is then assessed by the NIH Recombinant DNA Advisory Committee (RAC) to establish if it increases ethical, safety or medical concerns that contribute to more debate at one of the RAC’s public seminars (Ibraheem, Elaissari & Fessy, 2014).
Ethical Consequences of Gene Therapy
Gene therapy raises many ethical issues because it is involved in making changes to the body’s set of standard instructions. The ethical issues resulting from gene therapy. For instance, new gene therapy research has centralised on curing people by focusing the treatment to body tissue or cells such as blood cells or bone marrow. This particular gene therapy cannot be transferred to a person’s children. Thus gene therapy can be delivered to sperm and egg cells. This will enable the injected DNA to be moved on to future offsprings’. This procedure is referred to as germline gene therapy. There is a big controversy behind germline gene therapy. While it could be possible to save a family’s future generations from contracting a certain genetic disease, it might affect the growth of a fetus in unplanned ways or have continuous impacts that are yet to be proved. Individuals cannot choose whether to have the cure because persons who could be impacted by germline therapy are not yet born. As a result of this ethical concerns, the U.S Administration has banned the use of federal funds to carry out research on germline gene therapy in individuals.
Advancement in Gene Therapy
Recently, gene therapy is only been applied in research areas. The U.S Food and Drug and Administration (FDA) has not yet confirmed the usage of any gene therapy merchandise in America. Plenty of research studies has been done on gene therapy as a cure for cancer, HIV/AIDS, and generic conditions. Although the gene therapy is the only option for treating numerous diseases such as particular viral infection, and certain types of cancer. This procedure is still not safe, and research is ongoing to make it certain that it will be efficient and safe. Furthermore, gene therapy is only being used for the curing disorders that have no other choice of treatment.
In conclusion, gene therapy is a new procedure for curing numerous diseases such as viral infections, types of cancer, and genetic diseases. The process used involves the administration of specific DNA or RNA structure into the cell. Additionally, researchers are examining numerous methods to gene therapy. However, more research is still necessary to make sure the efficiency and safety of these procedures. Recently, gene therapy is being used to cure diseases where other therapies have been proved to be ineffective. Scientists are hoping that soon gene therapy will enable patients to be cured by injected the DNA into their cells instead of administering drugs or performing surgical procedures.
References
Brown, T. A., & Brown, T. (2016). Gene cloning and DNA analysis: an introduction. John Wiley & Sons.
Ibraheem, D., Elaissari, A., & Fessi, H. (2014). Gene therapy and DNA delivery systems. International journal of pharmaceutics, 459(1), 70-83.