Synp88, a promoter for the expression of genes

Abstract

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1800 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.

1 - 12 . (canceled) 
     
     
         13 . An expression cassette comprising an isolated nucleic acid molecule, the isolated nucleic acid molecule comprising the nucleic acid sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule is operatively linked to at least a nucleic acid sequence encoding for a gene, said nucleic acid sequence encoding for a gene being heterologous to the isolated nucleic acid molecule 
     
     
         14 . The expression cassette of  claim 13 , wherein said isolated nucleic acid molecule is effective to drive expression of the gene in a cell, wherein the cell is selected from the group consisting of a rodent cell, a non-human primate cell, and a human cell. 
     
     
         15 . The expression cassette of  claim 13 , further comprising a minimal promoter. 
     
     
         16 . The expression cassette of  claim 15 , wherein said minimal promoter comprises the nucleic acid sequence of SEQ ID NO:2. 
     
     
         17 . The expression cassette of  claim 13 , wherein the product of the gene is selected from the group consisting of a light-sensitive molecule, MT-ND4, MT-ND1, MT-ND6, MT-CYB, MT-CO3, MT-NDS, MT-ND2, 5 MT-COI, MT-ATP6, MT-ND4L, OPA1, OPA3, OPA7, ACO2, GDNF, CNTF, FGF2, BDNF, EPO, BCL2, BCL2L 1, endostatin, angiostatin, and sFlt, IL 10, IL 1 R1, TGFBI, IL4, and rod-derived cone viability factor (RdCVF). 
     
     
         18 . The expression cassette of  claim 17 , wherein the light-sensitive molecule is selected from the group consisting of a channelrhodopsin and a halorhodopsin. 
     
     
         19 . A vector comprising the expression cassette of  claim 13 . 
     
     
         20 . The vector of  claim 19 , wherein said vector is a viral vector. 
     
     
         21 . The vector of  claim 19 , wherein the vector is an adeno-associated virus (AAV) vector. 
     
     
         22 . A method of expressing a gene in a cell, the method comprising transfecting an isolated cell, a cell line, or a cell population with the expression cassette of  claim 13 . 
     
     
         23 . The method of  claim 22 , wherein the isolated cell is a rodent cell or the cell line or cell population comprises rodent cells. 
     
     
         24 . The method of  claim 22 , wherein the isolated cell is a non-human primate cell or the cell line or cell population comprises non-human primate cells. 
     
     
         25 . The method of  claim 22 , wherein the isolated cell is a human cell or the cell line or cell population comprises non-human primate cells. 
     
     
         26 . An isolated cell comprising the expression cassette of  claim 13 . 
     
     
         27 . The isolated cell of  claim 26 , wherein the expression cassette is stably integrated into the genome of said cell.