Everyone’s the remedial transmission of nucleic acid instead of

Everyone’s identity is influenced by their genes, which play a big factor in health, diseases, traits, and behavior. Starting in the 70’s, scientists have discovered ways to edit genetics in animals, and recently, to edit genetics in humans to cure diseases. As scientists learn more about genetic engineering, experts become increasingly worrisome about the ethical issues and the “what-ifs” in genetic engineering. When asked about their opinion on the subject, many people have similar responses as Marc Darnovsky, the executive director for the company CGS, who stated: “Although there may be some benefits from the use of the powerful new gene editing tools, they should not be used to create genetically-altered humans”. But what about human genetic engineering to cure diseases? Gene therapy? Many scientists understand the dangers of producing “designer babies”, yet scientists still do not know much about using gene therapy to treat diseases. Although there are still many situations in which genetic therapy has not been researched for, its significance is how many fields it is involved in. Human genetic engineering, the process of modifying the DNA in a genome, creates a medical treatment using gene therapy to cure or prevent diseases; however, its involvement has caused significant concerns over the moral implications of this technology, initiating the United States Department of Health to examine the laws and regulations on this technology.Human genetic engineering is the complex process of modification of the DNA in a genome. Gene therapy specifically is a form of treatment which is the remedial transmission of nucleic acid instead of a drug into a patient’s cell in order to treat their disease. In order to perform gene therapy, doctors require the patient’s DNA samples to be directed towards the flawed cells which then expresses or disrupts genes. In other words, since the proteins that are encoded in genes governs the way they function, by changing the levels of protein, the synthetic and degradative pathways are then affected, a process which they call gene expression. Doctors also use a capable method called gene disruption (also known as a gene knockout), where a working gene is replaced with a completely non functioning one through using recombinant DNA technology. The mutant phenotype gene’s biological function is then unveiled after a gene disruption. This technique is preferable compared to random mutagenesis since precise cells can be knocked out which then leaves all other genes unscaved from the mutagenic process. Researchers are also experimenting with replacing a mutated gene with a gene in perfectly good condition to eliminate or cure the spreading disease. Gene therapy is a rising hopeful treatment alternative, yet this technology still is considered extremely uncertain. Currently, the research on gene therapy is focused mainly on treating patients by aiming the therapy to body cells, and such cannot be passed down to the patient’s child. It could also be directed to an egg or sperm cells, resulting in the modified gene to then be inherited by the patients therapy, a method known as germline gene therapy. Studies show that many obstacles have been overcome and there has been major improvement since the first attempt of gene therapy done by Martin Cline in 1980, yet presently, majority of the research is only done for diseases without other alternatives. Martin Cline worked with two patients who suffered from hereditary blood disorders and operized a recombinant DNA (rDNA) transfer (also known as gene splicing) into their bone marrow cells. He researched at the University of California, Los Angeles and despite not obtaining proper permission from the International Review Board, he continued his work and consequently was forced to resign UCLA department chairmanship along with losing a number of research grants since he violated both the existing rDNA guidelines along with Federal regulations over human protection. In the Federal report, there was no suggestion that Cline had endangered the patient or that it was an unethical research. After all, both patients were alive; however, he has not given any evidence that his treatment was successful. Since then, many achievements have been made in genetic engineering and now, the complex process is being used to save lives.    Today genetic engineering is a groundbreaking technology used in many fields for numerous different purposes. Gene therapy specifically, has been successfully applied to produce replacement skin for a dying boy and will soon be used to help a young girl in the U.S fight a rare form of leukemia. Doctors in Europe constructed a new skin to save Hassan, a young boy suffering from junctional epidermolysis bullosa, a disease which induces immensely frail skin that is not only liable to infections and skin cancer, but it also blisters and tears handily. Such treatment was not used for the first time, but in fact, is used commonly to form skin grafts for burn victims. Although this form of gene therapy has been performed previously, Hassan’s treatment was easily the most advanced by having the most body surface (nine square feet) to be topped. Before receiving this treatment, the burn-unit tested everything: antibiotics, bandages, special nutritional measures, skin transplant from his father– all resulting in failure. In the end, doctors extracted a sample of Hassan’s skin and genetically engineered his cells, growing them into sheets of skin. Once transported back to Germany, doctors grafted the sheets of skin on Hassan, and in total, they replaced 80 percent of the boy’s skin. In the United States, the Food and Drug Administration (FDA) recently approved on August 30th, 2017 the first gene therapy which genetically modified the patient’s cells to fight cancer. This treatment known as Kymriah converts a patient’s cells into a “living drug,” training them to identify and infiltrate the disease, a rare form or leukemia. Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER) notes: “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.” Made by Novartis, this is  high-costing treatment of $475,000 which little can afford is usually used when all conventional options have failed. Scientists continue to explore curing and preventing diseases using gene therapy and according to the Alliance for Regenerative Medicine, 470 treatments are in initial clinical trials and another 34 in the final stages of testing imperative for the approval of the F.D.A.  People believe that gene therapy is an extremely promising technology in the future. Eric S. Lander refers to five diseases which he believes doctors could eradicate with gene therapy. He guesses that HIV, certain forms of genetic blindness, familial hypercholesterolemia, sickle-cell anemia, and hemophilia. Gene therapy is an extremely useful technique yet it is still risky and has much controversy around it, especially in the government. Genetic engineering’s engagement with stem cells and human embryos has caused compelling concerns over the moral implication of this technology, forcing the U.S. Health Department to examine the laws and regulations on such research.