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

Nature

Volumn 553, Issue 7687, 2018, Pages 217-221

  Gao, Xue ^a,b,c,j   Tao, Yong ^d,e,k   Lamas, Veronica ^d   Huang, Mingqian ^d   Yeh, Wei Hsi ^a,b,c   Pan, Bifeng ^f   Hu, Yu Juan ^d,e   Hu, Johnny H ^a,b,c   Thompson, David B ^a,b   Shu, Yilai ^d,g   Li, Yamin ^h   Wang, Hongyang ^d,i   Yang, Shiming ⁱ   Xu, Qiaobing ^h   Polley, Daniel B ^d   Liberman, M Charles ^d   Kong, Wei Jia ^e   Holt, Jeffrey R ^f   Chen, Zheng Yi ^d   Liu, David R ^a,b,c  

^a HARVARD UNIVERSITY   (United States)

^b HARVARD UNIVERSITY   (United States)

^c BROAD INSTITUTE OF MIT AND HARVARD   (United States)

^d MASSACHUSETTS EYE AND EAR INFIRMARY   (United States)

^e HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

^f BOSTON CHILDREN S HOSPITAL   (United States)

^g FUDAN UNIVERSITY   (China)

^h Tufts University   (United States)

ⁱ BEIJING UNIVERSITY OF POSTS AND TELECOMMUNICATIONS   (China)

^j Rice University   (United States)

^k SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CASPASE 9; CRISPR ASSOCIATED PROTEIN; LIPOSOME; MEMBRANE PROTEIN; TMC1 PROTEIN, MOUSE;

GENETIC ANALYSIS; GENOME; HAIR; HEARING; INHIBITION; LABORATORY METHOD; LIPID; MUTATION; RNA; RODENT;

ALLELE; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; AUTOSOMAL DOMINANT HEARING LOSS; BASE PAIRING; CELL SURVIVAL; CONTROLLED STUDY; EVOKED BRAIN STEM AUDITORY RESPONSE; FIBROBLAST; GENE; GENE DELIVERY SYSTEM; GENE DISRUPTION; GENE EDITING; GENE TARGETING; GENETIC VARIABILITY; HAIR CELL; HEARING IMPAIRMENT; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NEWBORN; NONHUMAN; PRIORITY JOURNAL; STARTLE REFLEX; TMC1 GENE; ANIMAL; AUDITORY STIMULATION; AUDITORY THRESHOLD; COCHLEA; COCHLEAR HAIR CELL; CRISPR CAS SYSTEM; CYTOLOGY; DISEASE MODEL; DOMINANT GENE; FEMALE; GENE THERAPY; GENETICS; HUMAN; MALE; METABOLISM; NUCLEOTIDE SEQUENCE; PATHOPHYSIOLOGY; PROCEDURES;

MUS;

ACOUSTIC STIMULATION; ALLELES; ANIMALS; ANIMALS, NEWBORN; AUDITORY THRESHOLD; BASE SEQUENCE; CELL SURVIVAL; COCHLEA; CRISPR-ASSOCIATED PROTEINS; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; EVOKED POTENTIALS, AUDITORY, BRAIN STEM; FEMALE; FIBROBLASTS; GENE EDITING; GENES, DOMINANT; GENETIC THERAPY; HAIR CELLS, AUDITORY; HEARING LOSS; HUMANS; LIPOSOMES; MALE; MEMBRANE PROTEINS; MICE; REFLEX, STARTLE;

EID: 85040458897     PISSN: 00280836     EISSN: 14764687     Source Type: Journal    
DOI: 10.1038/nature25164     Document Type: Article

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