Researchers have used gene editing to prevent mice from becoming deaf, the Guardian reports.
Harvard University's David Liu and his colleagues developed a CRISPR-Cas9 approach to treat mice with mutation in the Tmc1 gene that causes dominantly inherited hearing loss. As they report in Nature this week — and the Scan has more on it here — the researchers found that affected mice treated with their gene-editing construct had reduced hearing loss, increased hair cell survival, and lower auditory brainstem response thresholds.
Liu tells NPR that the difference between what treated and untreated ears could hear was "a normal quiet conversation" and "a garbage disposal."
Scientific American notes that as the difference between the affected and unaffected gene in this mouse is one base pair, the editing machinery had to be quite specific. Rather than use a viral vector, the researchers used a cationic lipid to deliver the Cas9 protein and guide RNA. Sciam says this delivery approach was more precise and enabled the machinery to quickly break down, limiting its effect on the unaffected allele.
The researchers say that a gene-editing approach like theirs may someday be able to help people with genetic deafness. "We're hopeful that our results will help guide the development of such strategies," says Liu at NPR.