摘要 |
<p>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 (See Figure 3). 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.</p> |