Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa

摘要

The present invention relates to a polynucleotide comprising a human photoreceptor-specific promoter element, a promoter including a core promoter, and at least one transgene. Furthermore, the present invention provides a plasmid containing the above polynucleotide, a viral vector containing the above polynucleotide, and a pharmaceutical composition containing the above polynucleotide. The invention also relates to said plasmid, said viral vector, or said pharmaceutical composition, for use as a medicament, in particular in the treatment of diseases of the retina. [Selection diagram] Fig. 7