| 摘要 |
Methods and compositions for the delivery and expression of a nucleotide sequence of interest to a cell of a mammalian eye, more particularly to a cell of the retina, more particularly to the retinal pigment epithelium (RPE) are provided. The methods and compositions of the present invention find use in modulating the expression of a nucleotide sequence of interest in a cell of a mammalian eye using the trans-viral vector system. The methods and compositions of the present invention find further use in the treatment and/or prevention of ocular disorders, particularly retinal degenerative disorders. The methods comprise administering to a retinal cell of a mammal in need thereof, a therapeutically effective amount of a trans-viral vector particle having a proviral genome comprising a nucleotide sequence of interest whose expression will lessen the clinical symptoms of the retinal disorder being treated.
|
