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expert reaction to study looking at using genome editing to restore hearing in deaf mice

Scientists have published a new study in Nature which reports the successful use of the CRISPR-Cas9 genome editing technique as a tool to correct hearing loss, in a mouse model of deafness.


Dr Simon Waddington, Reader in Gene Transfer Technology, UCL, said:

“I think the elegance of this study lies in the fact that the Cas9 protein and guide RNA themselves are delivered rather than expressed from a DNA vector. Therefore they present only transiently which confers on them a greater degree of safety. Nevertheless, the elimination of the dominant disease-causing allele by this method is permanent. This technology is particularly well-suited to genetic disease where precise, local delivery is required, in this case namely the cells within the cochlear of the inner ear. Is it an excellent illustration of how the toolkit available to the field of genetic medicine is expanding while the tools become ever more precise. Hitherto incurable and often even untreatable diseases are now within the scope of gene therapy.”


* ‘Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents’ by David R. Liu et al. published in Nature on Wednesday 20th December.


All our previous output on this subject can be seen at this weblink:


The SMC also produced a Factsheet / Briefing Note on genome editing which is attached and available here:


Declared interests

None received.

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