Chimeric promoter for cone photoreceptor targeted gene therapy

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United States of America Patent

PATENT NO 9375491
APP PUB NO 20140275231A1
SERIAL NO

14354471

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Abstract

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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.

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Patent Owner(s)

  • UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Boye, Sanford Leon Gainesville, US 4 31
Dyka, Frank Markus Gainesville, US 2 20
Hauswirth, William W Gainesville, US 44 652

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