Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents.
Gao X, Tao Y, Lamas V, Huang M, Yeh WH, Pan B, Hu YJ, Hu JH, Thompson DB, Shu Y, Li Y, Wang H, Yang S, Xu Q, Polley DB, Liberman MC, Kong WJ, Holt JR, Chen ZY, Liu DR.
Gao X, et al.
Nature. 2018 Jan 11;553(7687):217-221. doi: 10.1038/nature25164. Epub 2017 Dec 20.
Nature. 2018.
PMID: 29258297
Free PMC article.
Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1(Bth) allele into the cochlea of neonatal Tmc1(Bth/+) mice substantially reduced progressive hearing loss. We observed higher hair cell survival rates and lower auditory brainstem response thresholds in injected …
Injection of Cas9-guide RNA-lipid complexes targeting the Tmc1(Bth) allele into the cochlea of neonatal Tmc1(Bth/+) mice substantially reduc …