Nearly half of all hearing loss cases are due in some part to genetic factors. Researchers around the world, including many at Mass. Eye and Ear/Harvard Medical School, are hard at work on developing therapies to prevent hearing loss from genetic conditions.
In collaboration with the Broad Institute of MIT, Dr. Zheng-Yi Chen’s lab is working on an approach using the genome-editing technology CRISPR-Cas9. This week, they published some exciting results in the journal Nature. The research team successfully prevented mice with a particular mutation from developing hearing loss.
The therapy knocks out the defective gene by delivering a protein complex directly into the sound-sensing cells of the inner ear (known as “hair cells”), disrupting a mutation that would otherwise cause the cells to die. This approach differs from gene therapy, which delivers a correct copy to the cells to compensate for the defective gene.
“This is an exciting study that demonstrates the feasibility of a DNA-free, virus-free genome editing strategy for a type of autosomal dominant hearing loss characterized by progressive hair cell loss,” said Dr. Tina Stankovic, an otologic surgeon at Mass. Eye and Ear who was not involved with the study. “Augmenting the toolbox to treat genetic deafness is of major significance.”