Scientists develop treatment to prevent some deafness in mice

February 7, 2013

Hearing impairment is the most common sensory disorder, and scientists in North Chicago have recently discovered how to prevent it in mice holding a certain gene.

Usher syndrome is the leading genetic cause of combined deafness and blindness in humans. 

Dr. Michelle Hastings and her team from Rosalind Franklin University in North Chicago worked with researchers at Louisiana State University to correct the condition.

They developed a treatment using a synthetic molecule, called an antisense oligonucleotide, or ASO.

Hastings says a small dose was injected into newborn mice that have the Usher gene.

"They simply go into the cell and they recognize the mutation and blocks the mutation that causes the disease, correcting it and rescuing hearing in the mice that represent Usher syndrome," she said.

The ASO was designed to identify and fix the mutation in a gene called USH1C, the gene that causes Usher syndrome in humans. Hastings says the molecule was affective for mice that were injected within three to 13 days from birth.

The mice were able to hear at levels similar to non-Usher mice for about six months, nearly a third of their life span. 

"It was very surprising that it would get to the ear at that time and rescue for such a long time. It had a very lasting affect," Hastings said.

The molecule also corrected balance problems associated with the Usher gene, and Hastings believes it could be effective on blindness.

Hastings says it’ll be awhile before the treatment can be tested on humans. She says hearing develops for mice after they're born, but for humans, that happens in utero. So researchers would likely have to develop a suitable treatment before birth.

The findings were published this week in the journal Nature Medicine.