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.