Gene editing staves off deafness in mice

By Heidi Ledford

Genome editing has been used to reduce hearing loss in ‘Beethoven’ mice, which carry a mutation that causes deafness in both mice and humans.

The research relies on a technique called CRISPR–Cas9 to knock out a mutant form of the gene Tmc1. In doing so, it lays out a potential pathway for treating other genetic causes of hearing loss. It also addresses a major problem facing the field of genome editing: how to deliver the protein and RNA needed for the CRISPR–Cas9 technique into the cells of a living animal.

In this case, the researchers encapsulated the CRISPR components in positively charged fatty molecules called lipids, which are capable of crossing cell membranes. They then injected those particles directly into the inner ears of the mice, where the lipids were taken up by the hair cells that sense acoustic vibrations. The results are reported this week in Nature (X. Gao et al. Nature; 2017).

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