The treatment occurs by delivering a working version of the otoferlin gene to the inner ear. Otoferlin is a protein necessary for hearing, and a mutation in this gene can cause deafness. The gene therapy is delivered through a single injection into the cochlea, the spiral-shaped chamber of the inner ear containing hair cells.
The results of the research were published in the scientific journal Lancet. Children receiving gene therapy could hear sounds at a level of 45 decibels, the level of a normal conversation or the hum of a refrigerator. This is a significant improvement over the previous hearing level of 95 decibels, which was as loud as a food processor or motorcycle.
The researchers now plan to expand the study to include more patients and continue monitoring their results over a longer period of time. They are also working to develop a higher dose of gene therapy that could restore a more typical range of hearing.
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Source: Ferra
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