摘要:
The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated inwardly rectifying potassium channel (GIRK), in particular GIRK4 S143T, activated by G proteins recruited by cone opsin expressed in degenerating cones.
摘要:
The present invention relates to a nucleic acid sequence derived from the regulatory region of the human gamma-synuclein gene and having a promoter activity in retinal ganglion cells. The present invention also relates to expression cassettes or vectors comprising said promoter operably linked to a nucleic acid sequence encoding a polypeptide of interest as well as viral particles or host cells comprising said expression cassette or vector. The present invention also relates to the use of said expression cassettes, vectors, viral particles or cells in the treatment of ocular disease, in particular ocular disease associated with retinal ganglion cell or photoreceptor cell degeneration.
摘要:
Provided herein are methods for expressing a polynucleotide of interest in the retina of a subject. In particular, a method for expressing a polynucleotide of interest in the retina of a subject comprising the step consisting of injecting into the vitreous an amount of a vector containing the polynucleotide of interest in combination with an amount of an inhibitor of Dp71 expression is provided.