Reverse hearing loss!

Hearing loss is related to mutations in at least 100 different genes, but up to 16% of hereditary hearing loss can be traced back to only one gene, STRC, the second most common genetic cause

This technology can also be used to treat other conditions where the gene is very large, said Dr.

The team will now test whether this technology is applicable to human stereopsis genes, and test human inner ear cells from STRC hearing loss patients in a petri dish

Restore contact

In order for sound to be heard, the sensory hair cells of the inner ear must be in contact with the ear cover membrane, which vibrates the sound, and then converts these vibrations into signals and sends them to the brain

Holt said: "If stereocilin has a mutation, you don't have this contact, so the hair cells are not properly stimulated

Create a new gene therapy

In order to provide a healthy steroidal cilin gene, the research team used a synthetic adeno-associated virus (AAV) that is effective on hair cells

"The challenge we face is that the gene of stereopsis is too large to be integrated into the gene therapy vector," Holt said

The first author of this study, Dr.

Shubina-Oleinik said: "We later realized that there is a short amino acid at the beginning of a protein, and it acts like an'address' to guide the protein to the appropriate location in the cell

When they added the signal to the two halves of the protein, the two halves of the protein successfully joined together

Hearing recovery

The researchers used two types of hearing tests: one is similar to that of babies, and the other uses electrodes on the scalp to measure the response of the auditory brainstem to a range of sound frequencies and intensities

Shubina-Oleinik said: "The results are remarkable.

Shubina-Oleinik and Holt have filed a patent application for gene therapy technology

"It turns out that STRC gene variants are more common than we thought, which makes gene therapy very important for this disease," said Shearer, who worked with the Children's Rare Disease Cohort Initiative to screen a large number of The STRC mutant genome data set

The research was supported by IDDRC (approval number.

1U54HD090255), the Rare Disease Cohort Project of Boston Children's Hospital, the Jeffrey and Kimberly Barber Gene Therapy Research Fund, the Usher Syndrome Association and the Pour L'Audition Foundation
.
Holt is the scientific founder of Audition Therapeutics and an advisor to several biotech companies focusing on inner ear therapy.
He is also the inventor of the AAV9-PHP patent
.
B indicates that the gene is passed to the inner ear
.
The authors declare no other conflicts of interest
.

Article title

Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss