首页> 外国专利> OPTOGENETICALLY TRANSFORMED PHOTORECEPTOR PRECURSOR CELLS FOR THE USE IN THE TREATMENT OF RETINAL DEGENERATIVE DISEASES

OPTOGENETICALLY TRANSFORMED PHOTORECEPTOR PRECURSOR CELLS FOR THE USE IN THE TREATMENT OF RETINAL DEGENERATIVE DISEASES

机译:光学转化的光感受器前体细胞,用于治疗视网膜退行性疾病

摘要

The present invention relates to photoreceptor precursor cells comprising a heterologous nucleic acid encoding an optogenetic inhibitor. The present invention also relates to a pharmaceutical composition comprising photoreceptor precursor cells of the invention and a pharmaceutically acceptable excipient. The present invention relates to photoreceptor precursor cells or pharmaceutical composition of the invention for use in the treatment of a retinal degenerative disease, preferably a retinal degenerative disease related to a loss of function or death of photoreceptors. Finally, the present invention relates to a method for producing the photoreceptor precursor cells of the invention, comprising i) providing photoreceptor precursor cells; and ii) introducing into said precursor cells a nucleic acid encoding optogenetic inhibitor.
机译:本发明涉及包含编码光遗传学抑制剂的异源核酸的光感受器前体细胞。本发明还涉及包含本发明的感光体前体细胞和药学上可接受的赋形剂的药物组合物。本发明涉及本发明的感光体前体细胞或药物组合物,其用于治疗与视网膜功能丧失或死亡有关的视网膜退行性疾病,优选视网膜退行性疾病。最后,本发明涉及一种用于生产本发明的感光体前体细胞的方法,其包括:i)提供感光体前体细胞; ii)将编码光遗传学抑制剂的核酸引入所述前体细胞。

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