Previously in unit 6 of this class, we learned that gene therapy is the “introduction of genes into an afflicted individual for therapeutic processes” (Reece, p. 428). It revolves around the idea that a normal allele of the nonfunctional gene is inserted into the somatic cells of the tissue that is affected by the disorder. This procedure is commonly used to treat a variety of diseases today. Within the past couple of years, a group of scientists conducted a study to restore hearing in deaf mice using gene therapy in hopes of possibly then being able to perform this procedure on deaf children. The goal of this study was to conduct a procedure to find a more beneficial way to treat those with hearing loss. They used a gene therapy vector called the Anc80, which delivered the corrected gene into the mouse. The mice were studied and contained a mutation called Ush1c, which causes the protein harmonin to be ineffective and the sensory hair cell bundles that receive sound decline. A corrected Ush1c was injected into the inner ears of the mice, and it caused those sensory hair cell bundles to produce normal harmonin. They formed bundles which helped the brain receive signals and respond to sound waves.
The deaf mice in this study that were treated began to hear again. They have been tested and proven themselves to be able to respond to different types of sudden loud noises, and even responded to whispering. This procedure would be great to help treat Usher Syndrome patients, which is a disorder that can affect both hearing and sight. Today, Cochlear implants are the common procedure that is performed for those with the syndrome, but this technique using gene therapy can help children at a young age with their learning and speaking abilities into their adult lives. It seems like this would be most beneficial to begin this type of therapy early in the child’s life before they develop speech. This gene therapy procedure also can give people the ability to use their own sense of hearing, without the electronic device from the cochlear implant.
As cited in the Science Daily, Gwenaelle Geleoc who led this study says that “this strategy is the most effective one we’ve tested” (“Gene Therapy”, 2017). This procedure could impact many children in so many ways. These scientists achieved and found a procedure that works when it comes to deafness in humans. This article relates to what we read in Chapter 20 of our textbook, and it helped me further understand the concept of gene therapy in humans. Section 4 of Chapter 20 discusses how biotechnology, such as gene therapy, affects our lives in many ways. In addition, it exposed me to different disease treatments. The usage of genes in medical procedures is something I found interesting after this unit, and this article caused me to further explore this section of genetics.
- Boston Children’s Hospital. (2017, February 6). Gene therapy restores hearing in deaf mice, down to a whisper: Improved delivery vector better penetrates the inner ear, also restores balance in a mouse model of Usher syndrome. ScienceDaily. Retrieved February 6, 2017 from http://www.sciencedaily.com/releases/2017/02/170206111804.htm
- Reece, Jane. Campbell Biology AP Edition. 10th ed. Pearson