专利名称 | OPTOGENETICALLY TRANSFORMED PHOTORECEPTOR PRECURSOR CELLS FOR THE USE IN THE TREATMENT OF RETINAL DEGENERATIVE DISEASES | ||
申请号 | US16335700 | 申请日 | |
公开(公告)号 | US20200032203A1 | 公开(公告)日 | |
申请(专利权)人 | SORBONNE UNIVERSITE; CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE; INSERM (INSTITUT NATIONAL DE LA SANT?ET DE LA RECHERCHE MEDICALE) | 发明人 | JENS DUEBEL; MARIUS ADER; ANTOINE CHAFFIOL; MARCELA GARITA HERNANDEZ; MARUSA LAMPIC; DENIZ DALKARA; OLIVIER GOUREAU; JOS?ALAIN SAHEL |
专利来源 | 国家知识产权局 | 转化方式 | |
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. |
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