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ARTIFICIAL TRANSCRIPTION FACTORS FOR THE TREATMENT OF DISEASES CAUSED BY OPA1 HAPLOINSUFFICIENCY
专利权人:
ALIOPHTHA AG
发明人:
Albert NEUTZNER,Josef FLAMMER,Alice HUXLEY
申请号:
US14781710
公开号:
US20160039893A1
申请日:
2014.04.02
申请国别(地区):
US
年份:
2016
代理人:
摘要:
The invention relates to an artificial transcription factor comprising a polydactyl zinc finger protein targeting specifically the OPA1 promoter fused to an activatory protein domain, and a nuclear localization sequence. Artificial transcription factors directed against the OPA1 promoter are useful for the treatment of diseases associated with OPA1 haploinsufficiency, such as autosomal dominant optic atrophy, syndromic autosomal dominant optic atrophy plus and normal tension glaucoma.
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中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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