Gene Therapy Brings Hearing to Kids With Congenital Deafness

    The article titled “Gene Therapy Brings Hearing to Kids With Congenital Deafness” reports that gene therapy is being tested to bring back the hearing of children with congenital deafness. This is done by injecting the protein responsible for transferring sound singles called otoferlin, into the inner ear of six deaf children, and studies have found that they have experienced hearing within approximately six months of the injection. A lack of otoferlin is typically caused by a gene mutation that ends up making people deaf from birth. Otoferlin correction is a groundbreaking study because it is the first treatment in almost 60 years for deafness. Gene therapy for deaf children has also correlated to better speech patterns and allowed children to carry out thorough conversations. 

    I think this article is great and brings a lot of meaningful insight into how long there has been no treatment or cure for congenital deafness. It shows that research with gene therapy is also advancing to a point where researchers are making groundbreaking discoveries for new treatments for different disabilities or diseases. 

Gene Therapy Reverses Congential Deafness in Mice

Scientists from the Institut Pasteur, Inserm, CNRS, and many other universities have collaborated to restore hearing in adult mice of DFNB9 deafness. Someone who has DFNB9 deafness are deficient in the gene coding for otoferlin, which is a protein that transmits sound information. The scientists restored the auditory synapase threshold, which receives the sound information, to a near-normal level. DFNB9 deafness is the same for humans and mice, that is why mice are used as models to test the efficiency of gene therapy.  Scientists came up with a Adeno-associated virus (AAV) approach, known as the dual AAV strategy becuase it uses two different recombinant vecotrs. An intracochlear injection of the vector pair was injected in adult mice to reconstruct the otoferlin coding region, resulting to a long-term restoration of otoferlin. With these findings, scientists can help individuals with DFNB9 deafness, to restore their hearing and also discover other types of deafness and hopefully be able to cure the other types of deafness.

 

I found this article to be interesting because a small percentage of individuals suffer with DFNB9 deafness since the prenatal stages, so they are born with it. It is interesting how scientists were able to use mice as a model for DFNB9 deafness since humans and mice express the mutation the same way. The scientists were able to restore deafness in the mice models for log-term and hopefully, it will restore hearing for humans long-term as well. Also, with these findings, scientists can also discover other types of deafnesses associated with humans and mice and hopefully find a cure for those other types of deafnesses.