(CTN News) – Adeno-associated virus Gene Therapy vectors have been successfully used by researchers at Mass Eye and Ear, a part of Mass General Brigham Hospital, in gene therapies as a means of treating Gene Therapy hearing loss in aged mouse models with adeno-associated virus vectors.
As a result of this breakthrough, we think that similar therapies could be effective for treating genetic hearing loss in the elderly population of the human race as well.
Approximately one in ten people worldwide will suffer from hearing loss by 2050, according to the World Health Organization. Worldwide, there are hundreds of millions of cases of hearing loss each year, but genetic hearing loss is often the most difficult to treat.
Though hearing aids and cochlear implants offer limited relief from this group of genetic conditions, there is no available treatment that can reverse or prevent them, prompting scientists to investigate gene therapy as an alternative treatment option.
Adeno-associated virus (AAV) vectors have been one of the most promising tools used in these treatments in recent years, which has galvanized the hearing-loss community in recent years.
In spite of the fact that the vectors have achieved success in restoring hearing in neonatal animals with genetic defects, they have yet to demonstrate this ability in mature or aged animal models.
Since all humans are born with fully developed ears, this proof-of-concept must be carried out before any human beings with genetic hearing loss can be tested with this intervention.
An AAV vector has recently been successfully demonstrated in aged animal models for the first time by Mass Eye and Ear, a member of Mass General Brigham, through the development of a mouse model containing a mutation similar to the TMPRSS3 human Gene Therapy defect that results in progressive hearing loss.
After injecting the animals with an AAV carrying a healthy human TMPRSS3 gene, researchers reported in the journal Molecular Therapy that robust hearing rescue was observed in the aged mice.
This study suggests that virally mediated gene therapy, whether used alone or in conjunction with a cochlear implant, may be an effective means of treating genetic hearing loss, according to corresponding author Zheng Yi Chen, a doctorate in otolaryngology at Mass Eye and Ear Research Institute.
“This was also the first study that was able to rescue hearing in mice that were aging, which indicates that DFNB8 can be used as a treatment for DFNB8 patients at any age.
It also demonstrates the feasibility of other Gene Therapy in the elderly population by demonstrating the feasibility of the study.”