Molecule Drug Therapy and Progressive Hearing Loss

A step closer to small molecule drug therapy in humans to combat hereditary progressive hearing loss.

One might think that once the cause for genetic, progressive hearing loss, like DFNA27 is identified, targeted therapy would swiftly follow. In actuality, the infinite and somewhat incomprehensible smallness associated with molecular research is akin to finding a needle in a haystack, with the haystack being DFNA27.

Researchers at the University of Iowa and the National Institute on Deafness and Other Communication Disorders report that hearing was restored, though not fully, in genetically altered “exon-4 deficient” mice . These mice were bred with an active “REST” gene, located in the DFNA27 region and served as a model for those with DFNA27 progressive deafness. Researchers applied a small molecule “blocking process” to turn off REST, which improved hearing in the test mice.

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References

Nakano Y. (2018) Defects in the Alternative Splicing-Dependent Regulation of REST Cause Deafness. Cell. June 8.

National Institutes of Health. (2018) Novel Drug Therapy Partially Restores Hearing in Mice. June 28.