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ARTIFICIAL TRANSCRIPTION FACTORS FOR THE TREATMENT OF DISEASES CAUSED BY OPA1 HAPLOINSUFFICIENCY
ARTIFICIAL TRANSCRIPTION FACTORS FOR THE TREATMENT OF DISEASES CAUSED BY OPA1 HAPLOINSUFFICIENCY
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机译:人工转录因子用于治疗 OPA1 I>肝功能不全所致疾病
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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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