Gene Therapy Restores Deaf Mice Hearing
Through gene therapy, a genetically engineered deaf mice's hearing was restored. The team who has successfully conducted the experiment is now considering restoring a much higher level of hearing, if possible, down to 25 decibels using a more improved gene therapy vector developed at the Massachusetts Eye and Ear.
Science Daily published a summary of the papers posted in Nature Biotechnology. The study revealed a new synthetic vector, Anc80, which managed to insert a gene into the difficult to reach outer hair cells of the cochlea of a mouse. Experts included in the experiment include Jeffrey R. Holt Ph.D., of Boston Children's Hospital; Luk H. Vandenberghe, Ph.D., who led Anc80's development in 2015 at Mass. Eye and Ear's Grousbeck Gene Therapy Center and Konstantina Stankovic, MD, Ph.D., of Mass. Eye and Ear.
Findings of the experiment revealed that the Anc80 managed to infect cells in the inner ear of the mouse, opening the possibilities of using the same method to human patients. Anc80 was also used on a corrected gene of a mouse subject with Usher syndrome, a common genetic form of deaf-blindness that also impairs the balancing function of the mouse. The outer hair cells amplified the sound which allowed the inner hair cells in the cochlea send stronger signals to the brain, thus hearing.
Daily Mail UK reports that this technique can be effectively used in humans in the next three years. Additional research and testing will be needed to be able to create a technology that would be safe and effective to use for human patients.
The initial experiment wherein the mice were treated straight after birth showed positive results while delaying it by 10 to 12 days, created failed results. The experiment was mainly conducted to restore hearing problems. However, gene therapy is also being explored for its potential in treating eye disorders.
The future of hearing gene therapy becomes brighter with this presently reported success on mice subjects. Further investigations on the possible reactions of humans to this new vector are being conducted to explore the possibilities of equipping such methods in treating human hearing and balance problems.