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. Author manuscript; available in PMC: 2019 Aug 18.
Published in final edited form as: N Engl J Med. 2018 Mar 29;378(13):1255–1256. doi: 10.1056/NEJMcibr1716789

Figure 2. Use of CRISPR-Cas9 in Knockdown of Mutant Tmc1 Allele to Restore Hair-Cell Function.

Figure 2.

Shown is the application of genome editing to the Beethoven mouse model of hereditary hearing loss. A guide RNA is designed to target the mutant Tmc1 allele and recruits Cas9 endonuclease to make a double-stranded cut. Random insertions and deletions are incorporated into the cut, and the mutant Tmc1 allele is inactivated.