Gene-Editing Complex Restrains Hearing Loss in Animal Model

Gene Editing Complex Restrains Hearing Loss in Animal Model

Scientists have developed a CRISPR-Cas9 gene-editing complex, which can be delivered directly into hair cells of inner ear to prevent hearing loss

Researchers from the Broad Institute of MIT and Harvard delivered the complex directly into the hair cells of the inner ear in a mouse model of human progressive genetic progressive deafness, which inactivated a gene known to function for hearing loss. The study was published in Nature Journal on December 22, 2017.

Hearing loss is common type of sensory loss in humans and it is usually caused due to genetic element. Inner ear hair cells convert sound wave vibrations into electrical signals, which are interpreted by brain. Genetic mutation in the TMC1 gene results into production of toxic protein by hair cells that accumulates in cells and inhibit their function. The mutation causes progressive hearing loss in individuals resulting into deafness.

By transferring CRISPR-Cas9 gene editing complex to the mutated gene researchers were able to preserve the inner ear hair cells in mice that were otherwise genetically destined to become deaf. The gene-editing mix was injected into the cochlea of new-born mice with the TMC1 gene mutation. If the mice was left untreated, it would have experienced hearing loss at four weeks of age leading to deafness at eight. At four weeks, mice that were not given the treatment had a detectable brainstem response to sounds of around 80 decibels and mice that were given the treatment had responses to sound starting at around 65 decibels.

“These results inform the potential development of a treatment for a subtype of genetic hearing loss, but making sure the method is safe and effective is critically important before we propose moving closer to human trials,” said co-senior author Liu. Physiological measurements showed that the treated mice had a higher rate of inner ear hair cell survival than untreated mice and genetic sequencing of the edited cells showed that the TMC1 gene had successfully been disabled in 94% of cases.

According to Hearing Aids Market report published by Coherent Market Insights, Sensorineural hearing loss is a permanent type of hearing loss caused due to damage to the inner cells or auditory nerve of the ear, which can be treated with gene editing therapy. The team now intends to develop the treatment in larger animal models of genetic progressive hearing loss.

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