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GENE THERAPY FOR THE TREATMENT OF CNGB1-LINKED RETINITIS PIGMENTOSA
GENE THERAPY FOR THE TREATMENT OF CNGB1-LINKED RETINITIS PIGMENTOSA
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机译:基因疗法治疗CNGB1连锁性视网膜色素沉着症
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摘要
The present invention relates to a polynucleotide comprising a promoter comprising a human photoreceptor-specific promoter element, a core promoter and at least one transgene. Further, the invention provides a plasmid comprising the polynucleotide, a viral vector comprising the polynucleotide and a pharmaceutical composition comprising the polynucleotide. The invention also relates to the plasmid, the viral vector or the pharmaceutical composition for use as a medicament, in particular for use in the therapy of diseases of the retina.
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