The Board of Regents of the University of Oklahoma
发明人:
Jian-Xing Ma,Yusuke Takahashi,Gennadiy Moiseyev
申请号：
US15500837
公开号：
US10494619B2
申请日:
2015.07.27
申请国别(地区):
US
年份:
2019
代理人:
摘要：
Mutant mammalian RPE65 proteins and portions thereof, and nucleic acids encoding the mutants, for use in treating a condition related to retinal degeneration in a subject, the mutant mammalian RPE65 proteins or portions thereof having isomerohydrolase activity. A gene therapy method of treating a condition related to retinal degeneration in a mammalian subject in need of such treatment, comprising administering to the subject a therapeutically-effective amount of a vector comprising a nucleic acid encoding a mutant mammalian RPE65 protein or a portion thereof. A method of treating a condition related to retinal degeneration in a subject in need of such treatment, comprising administering to the subject a therapeutically-effective amount of at least one of a mutant mammalian RPE65 protein or a portion thereof.
