US2022186214A1PendingUtilityA1

Promoter for the expression of genes

Assignee: FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESPriority: Oct 14, 2015Filed: Jan 7, 2022Published: Jun 16, 2022
Est. expiryOct 14, 2035(~9.2 yrs left)· nominal 20-yr term from priority
A61K 48/0058C12N 15/86C07K 14/515C12N 15/85C12N 2830/008C12N 15/11
67
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Claims

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 600 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 endothelial cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.

Claims

exact text as granted — not AI-modified
1 - 12 . (canceled) 
     
     
         13 . An isolated nucleic acid molecule comprising the nucleic acid sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule is effective to drive expression of a gene in a cell when a nucleic acid sequence coding for said gene is operatively linked to said isolated nucleic acid molecule. 
     
     
         14 . The isolated nucleic acid molecule of  claim 13 , further comprising a minimal promoter 
     
     
         15 . The isolated nucleic acid molecule of  claim 14 , wherein the minimal promoter comprises the nucleic acid sequence of SEQ ID NO:2. 
     
     
         16 . The isolated nucleic acid molecule of  claim 13 , wherein the cell is a rodent cell. 
     
     
         17 . The isolated nucleic acid molecule of  claim 13 , wherein the cell is a human cell. 
     
     
         18 . The isolated nucleic acid molecule of  claim 13 , wherein the product of the gene is an agent controlling vasculogenesis and/or angiogenesis. 
     
     
         19 . The isolated nucleic acid molecule of  claim 18 , wherein the agent controlling vasculogenesis and/or angiogenesis is a VEGF-inhibitor. 
     
     
         20 . An expression cassette comprising the isolated nucleic acid molecule according to  claim 13 , wherein said isolated nucleic acid molecule is operatively linked to at least a nucleic acid sequence encoding for a gene. 
     
     
         21 . The expression cassette of  claim 20 , wherein the cell is a rodent cell. 
     
     
         22 . The expression cassette of  claim 20 , wherein the cell is a human cell. 
     
     
         23 . A vector comprising the expression cassette of  claim 20 . 
     
     
         24 . The vector of  claim 23 , wherein said vector is a viral vector. 
     
     
         25 . The vector of  claim 24 , wherein the viral vector is an adeno-associated virus (AAV) vector. 
     
     
         26 . 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 20 . 
     
     
         27 . The method of  claim 26 , wherein the isolated cell is a rodent cell, or the cell line or the cell population comprises a rodent cell. 
     
     
         28 . The method of  claim 26 , wherein the isolated cell is a human cell, or the cell line or the cell population comprises a human cell. 
     
     
         29 . An isolated cell comprising the expression cassette of  claim 20 . 
     
     
         30 . The isolated cell of  claim 29 , wherein the expression cassette is stably integrated into the genome of said cell. 
     
     
         31 . A kit for expressing a gene in a cell, the kit comprising the isolated nucleic acid molecule of  claim 13 . 
     
     
         32 . The kit of  claim 31 , further comprising a needle, a syringe, or instructions for administering the isolated nucleic acid molecule.

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