Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

• Published in: Nature (London) Vol. 553; no. 7687; pp. 217 - 221
• Main Authors: Gao, Xue, Tao, Yong, Lamas, Veronica, Huang, Mingqian, Yeh, Wei-Hsi, Pan, Bifeng, Hu, Yu-Juan, Hu, Johnny H., Thompson, David B., Shu, Yilai, Li, Yamin, Wang, Hongyang, Yang, Shiming, Xu, Qiaobing, Polley, Daniel B., Liberman, M. Charles, Kong, Wei-Jia, Holt, Jeffrey R., Chen, Zheng-Yi, Liu, David R.
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 11.01.2018; Nature Publishing Group
• Subjects: 45; 45/41; 631/1647/1511; 631/61/2297; Acoustic startle response; Acoustic Stimulation; Alleles; Animals; Animals, Newborn; Auditory Threshold; Autosomal dominant inheritance; Base Sequence; Brain stem; Care and treatment; Cell Survival; Cochlea; Cochlea - cytology; Cochlea - metabolism; CRISPR; CRISPR-Associated Proteins - administration & dosage; CRISPR-Associated Proteins - metabolism; CRISPR-Associated Proteins - therapeutic use; CRISPR-Cas Systems; Deafness; Deoxyribonucleic acid; Disease Models, Animal; DNA; Ears & hearing; Evoked Potentials, Auditory, Brain Stem; Female; Fibroblasts; Gene Editing - methods; Gene therapy; Genes; Genes, Dominant - genetics; Genetic disorders; Genetic engineering; Genetic factors; Genetic Therapy - methods; Genome editing; Genomes; Hair Cells, Auditory - cytology; Hearing loss; Hearing Loss - genetics; Hearing Loss - physiopathology; Hearing Loss - prevention & control; Humanities and Social Sciences; Humans; letter; Lipids; Liposomes; Male; Membrane Proteins - genetics; Methods; Mice; multidisciplinary; Mutation; Neonates; Proteins; Reflex, Startle; Ribonucleic acid; RNA; Science; Survival

CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing. Hindering heritable hearing loss Nearly half of all deafness cases arise from genetic factors, yet there are limited treatmen...