Gene Therapy and its Potential to Cure Deafness
Losing a vital sense makes living life more difficult. Gene therapy, the process of replacing faulty genes with genes genetically engineered to replace them, can potentially cure deafness. Yashimo Raphael experimented with intentionally deafened guinea pigs and the gene Atoh 1, a gene said to replace lost hair cells in the inner ear. He found that hair cells grew, but were not fully functional. The slight aid in hearing the gene did give the guinea pigs almost completely disappeared after a few weeks time. Although the new hair cells did not function properly, the fact that they grew defied nature and was a successful start.
Deafness affects millions of people in the United States every year. Cochlear implants and hearing aids are two methods to treat the hearing impaired, but the person has to rely on the device to hear sounds. First announced in Nature Medicine, scientists at the University of Michigan Medical School have discovered a gene that could potentially cure deafness.
Gene therapy, a relatively new innovention, is becoming popular across the country. Gene therapy modifies a part of an organism, whereas cloning creates an entirely recreated organism. This technique can be conducted in vivo in either somatic or germ cells. The process is essentially aimed at fixing a genetic disorder or disease by inserting a functional gene to replace the faulty one (Houdebine 2003). Many methods to conduct a gene transfer have been tested. The two types are in vivo and in vitro. Transferring genes in vivo means placing the functional genes directly into the target tissue; while vitro transfers creates the genes outside of the body, in Petri dishes. Vitro is an expensive process that r...
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...to create a genetically perfect person is a controversial issue in the world genetically engineering. While Atoh 1 cannot currently cure hearing it can re-grow precious hair cells, making it valuable in the slow steps of finding an eventual cure.
References
Coghlan, A (2005, February) Gene Therapy is first deafness ‘cure.’ New Scientist. 7/25/05: www.newscientist.com
Houbebine, Louis-Marie. (2003). Animal transgenesis and Cloning. Chichester:
The Atrium
Maugh, T. (2005, February) Gene therapy fixes animal hearing. Los Angeles Times. www.detnews.com
Pau, H., r.w. Clarke. (2004). Advances in the genetic manipulations in the treatment of hearing disorders. Clinical Otolaryngology (29), Page 574
Pobojewski, S. (2005, February). U-M scientists use gene therapy to grow new hair cells and restore hearing in adult guinea pigs. University of Michigan Health Systems.
In support of Gallaudet University's, “Deaf President Now!” protest, Rev. Jesse L. Jackson once said “The problem is not that the deaf do not hear. The problem is that the hearing world does not listen.” (Lee) This concept been seen throughout Deaf history; Today it is especially noticeable now that technology has been developed to restore some hearing to many who are deaf. However these seemingly miraculous devices such as hearing aids and cochlear implants (CI) are a gilding for the real problem. CI’s in particular are thought to be a miracle cure for deafness, this misconception causes a great friction between Deaf and hearing culture. Deaf people should not be expected to get CI’s to “cure” their deafness for three primary reasons: CI’s promise more than they can deliver, they are an affront to Deaf culture, and ASL is a more effective way to insure development.
What I found most interesting about Jarashow’s presentation were the two opposing views: Deaf culture versus medical professionals. Within the Deaf culture, they want to preserve their language and identity. The Deaf community wants to flourish and grow and do not view being deaf as a disability or being wrong. Jarashow stated that the medical field labels Deaf people as having a handicap or being disabled because they cannot hear. Those who are Deaf feel as though medical professionals are trying to eliminate them and relate it to eugenics. It is perceived that those in that field are trying to fix those who are Deaf and eliminate them by making them conform to a hearing world. Those within the Deaf community seem to be unhappy with devices such
Genetic engineering, the process of using genetic information from the deoxyribonucleic acid (DNA) of cells to fix or improve genetic defects or maladies, has been developing for over twenty years. When Joseph Vacanti, a pediatric surgeon at Children’s Hospital, and Robert Langer, a chemical engineering professor at MIT, first met as researchers in the 1970’s, they had little knowledge of the movement they would help found. After they discovered a method of growing live tissue in the 1980’s, a new science was born, and it races daily towards new discoveries and medical breakthroughs (Arnst and Carey 60). “Tissue engineering offers the promise that failing organs and aging cells no longer be tolerated — they can be rejuvenated or replaced with healthy cells and tissues grown anew” (Arnst and Carey 58). The need for genetic engineering becomes quite evident in the promises it offers in various medical fields, as well to financial ones. Despite critics’ arguments about the morality or practicality of it, genetic engineering should continue to provide the essential benefits it has to offer without unnecessary legal impediment.
Gene therapy is a technique used in attempts to cure or prevent genetic diseases at the molecular level (basically at the source) by correcting what is wrong with defective genes, a good version of the defective gene is introduced into the existing cells. This technique is still considered experimental, only being done through clinical trials. This idea was first suggested during the 1950’s. The basic idea was “if the basses can be arranged incorrectly then why can’t they be rearranged in the correct way?”
Gene therapy is a provisional technique that is the insertion of normal genes into the cells where there is a missing or miscoded gene to fix a genetic disorder. In the 1960s and early 1970s,
5. Leiden, Dr. Jeffrey M. "Gene therapy -- Promises, Pitfalls, and Prognosis." The New England Journal of Medicine 28 September 1995: 871-873.
National Institute of Health. (2011). National Institute on Deafness and other communication disorders: Improving the lives of people who have communication disorders. National Institute on
Gene therapy is one of the most rapidly growing techniques in the medical field. One out of ten people are affected by genetic disorders. Defective genes that code for an incorrectly formed protein, resulting in a severely hindered function, cause genetic disorders or process that are usually lethal. The essential idea was to replace the defective genes causing the disorder by introducing a confirmed healthy form into the patient through some sort of vector. Vectors are fragmented down into two groups, viral and non-viral.
Every living thing is the product of the genes that were passed down from ancestors. Genes make up everything we are. One gets their traits from their parents. Most people live full lives with relatively good health. However, some people inherit mutated genes or faulty genes. This could lead to genetic disorders that could be life threatening. Even today, many genetic disorders still remain incurable, leaving many people without hope. Genetic therapy could be their answer. It is through this research that the cure for genetic disorders can be found. Though some people believe it is unethical or immoral to alter genes, current therapeutics have not been able to save the lives of the patients with these diseases. Genetic therapy for medicinal use is necessary because it will prove vital in saving lives of those stricken with a genetic disorder.
Gene therapy is considered to be a relatively new form of treatment that could eventually benefit people who have one of the thousands of diseases caused by a genetic disorder. Using gene therapy can treat, prevent or cure these types of diseases1. This “medicine of the future” has been worked on for decades but there is not a huge amount of success for human patients because gene therapy isn’t completely practical yet. Human clinical trials are still in the research phase because gene therapy can be unpredictable. Very few patients have received gene therapy. Researchers are working extremely hard to break through the challenges they are facing and make sure that gene therapy is safe and effective for humans.
Thus far, researchers have had minimal success in using gene therapy to correct most genetic conditions and no researcher has used gene therapy to correct genetic impairments in a fetus (Parens). Although it is impossible to correct genetic flaws, we have discovered how to test for over 400 conditions, from those viewed as severe, such as Tay Sachs, to those that many might describe as relatively minor, such as polydactyly (a trait involving an extra little finger) (Parens).
So today, I have shared with you my journey in deafness. Being deaf can be hard, but it is not the end of the world. I can do what anyone else can do such as talk, play sports and hang out with friends. Every person’s journey is different. For me the key to success is perseverance.
Johnston, Josephine, and Françoise Baylis. (2004). "What Happened to Gene Therapy? A Review of Recent Events." Clinical Researcher 4: 11-15.
Gene therapy employs vectors to deliver functioning genes into cells, a ‘vector’ being a vehicle in which DNA can be transferred. They can be viral or non-viral, and treatment can occur in vitro and ex vivo (inside and outside the body) (ScienceDaily, 2014; University of Utah, 2014). The use of viral vectors mimics the survival techniques of viruses. A virus is a miniscule particle containing genetic material; lacking material to reproduce on its own, it infects a host, injecting its DNA or RNA, and utilises the host’s cellular machinery to reproduce (Freudenrich, 2014). In gene therapy,...
Gene therapy poses many risks, but may prove the ideal solution for countless diseases. As seen throughout the past few decades, gene therapy can cure genetically inherited diseases by introducing therapeutic genes into the body. Critics may argue that there are moral and ethical problems associated with this novel technique, but for the most part scientists realize the importance this advancement will have. Gene therapy may be the key to curing dozens of diseases, and has endless possibilities, but more research is needed before its safe or accepted as common practice.