Massachusetts Eye and Ear Infirmary;Editas Medicine, Inc.
发明人:
PIERCE, Eric A.,MARGULIES, Carrie Marie,PENDSE, Nachiket D.,GLOSKOWSKI, Sebastian W.,LIU, Qin,MAEDER, Morgan L.
申请号：
AU2019237541
公开号：
AU2019237541A1
申请日:
2019.03.25
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
Compositions for use in treating subjects with USH2A-associated retinal and/or cochlear degeneration that result from mutations in exon 13 of the USH2A gene by deletion of exon 13 from the USH2A gene or transcripts, and methods of use thereof, as well as genetically modified animals and cells.
