Loading patent…
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsSanford Leon Boye, Frank Markus Dyka, William W. Hauswirth
Estimating the term…
The subject invention concerns materials and methods for providing for cone cell specific expression of a polynucleotide in a human or animal. One aspect of the invention concerns a polynucleotide promoter sequence that directs expression of an operably linked polynucleotide in cone cells. In one embodiment, a polynucleotide of the invention comprises a nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene that is positioned upstream of a promoter nucleotide sequence of a cone transducin alpha-subunit (GNAT2) gene. Another aspect of the subject invention concerns methods for expressing a selected polynucleotide in cone cells. The selected polynucleotide can be provided in a polynucleotide of the invention wherein the selected polynucleotide is operably linked to a polynucleotide promoter sequence of the invention. In one embodiment, the selected polynucleotide sequence is provided in a polynucleotide vector of the invention. The vector comprising the selected polynucleotide is then introduced into a cell. The selected polynucleotide is expressed only in cone cells, with very little, if any, expression in rods or other cells. A selected polynucleotide can be one that encodes, for example, a therapeutic protein or a functional protein that is defective or underexpressed in the targeted cone cells.
A polynucleotide that promotes expression in cone cells of an operably linked nucleotide sequence, wherein said polynucleotide comprises an enhancer nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene sequence that is positioned upstream of a promoter nucleotide sequence of a cone transducing alpha-subunit (GNAT2) gene sequence.
12-15. (canceled)
An expression construct comprising a polynucleotide that promotes expression in cone cells of an operably linked nucleotide sequence, wherein said polynucleotide comprises an enhancer nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene sequence that is positioned upstream of a promoter nucleotide sequence of a cone transducing alpha-subunit (GNAT2) gene sequence;
(canceled)
22-24. (canceled)
(canceled)
A method for treating or ameliorating a disease or condition associated with a cone photoreceptor in a human or animal eye, said method comprising administering to the person or animal a polynucleotide that promotes expression in cone cells of an operably linked nucleotide sequence, wherein said polynucleotide comprises an enhancer nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene sequence that is positioned upstream of a promoter nucleotide sequence of a cone transducing alpha-subunit (GNAT2) gene sequence;
31-44. (canceled)
Dependencies are inferred from the claim text.