CRISPR Treatment Prevents Hearing Loss in Mice with Genetic Deafness

Almost half of all deafness cases are contributed by genetic factors. However, effective targeting of these deafness associated genes has not been very successful. Recently, researchers from Harvard University and MIT employed CRISPR/Cas9 technology to genetically target dominant alleles that are associated with hearing loss.

In this study, Gao et al. injected Cas9-gRNA-lipid complexes, targeting the Tmc1 Beethoven allele, into the cochlea of neonatal mice with Tmc1 mutations and measured indicators of hearing loss. The results of this study showed that CRISPR/Cas9 treatment significantly reduced progressive hearing loss when compared to control group.

Overall, by using cationic lipid-mediated delivery method, CRISPR Cas9/sgRNA complexes can selectively disrupt deafness-associated dominant alleles in a mouse model with genetic deafness, therefore indicating a potential treatment strategy for autosomal dominant hearing loss in human.

• Gao et al. Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents. Nature. 2017 Dec. doi:10.1038/nature25164

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