Gene Therapy

CRISPR corrects deafness in mice

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Science  12 Jan 2018:
Vol. 359, Issue 6372, pp. 173-174
DOI: 10.1126/science.359.6372.173-e

Limited treatment options are available for individuals with hereditary hearing loss. CRISPR-Cas9 editing can be used as molecular scissors that snip out mutant DNA sequences to permit gene repair. Gao et al. asked whether the Cas9 cutting enzyme could be used to correct genetic deafness caused by dominant mutations in the Tmc1 gene. The researchers performed a lipid-mediated delivery of Cas9-RNA complexes to the inner ear of neonatal mice. They were able edit the mutant Tmc1 gene within the cochlear hair cells that sense acoustic vibrations. Mice showed signs of improved cochlear function and hearing restoration.

Nature 10.1038/nature25164 (2017).

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