US2021269826A1PendingUtilityA1

Promoter for the specific expression of genes in müller cells

Assignee: FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESPriority: Apr 30, 2015Filed: Feb 10, 2021Published: Sep 2, 2021
Est. expiryApr 30, 2035(~8.8 yrs left)· nominal 20-yr term from priority
C12N 2840/007C12N 2750/14143C12N 15/86C12N 15/85C07K 14/705C12N 5/0621
65
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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 400 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 Müller 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 . An isolated nucleic acid molecule comprising a nucleic acid sequence of at least 400 base pairs and having at least 80% identity to the nucleic acid sequence of SEQ ID NO:1. 
     
     
         2 . The isolated nucleic acid molecule of  claim 1 , further comprising a minimal promoter. 
     
     
         3 . (canceled) 
     
     
         4 . An expression cassette comprising:
 the isolated nucleic acid molecule according to  claim 1 ; and   a nucleic acid sequence coding for a gene, wherein said isolated nucleic acid molecule is operatively linked to the nucleic acid sequence encoding for a gene.   
     
     
         5 . A vector comprising the expression cassette of  claim 4 . 
     
     
         6 . The vector of  claim 5 , wherein said vector is a viral vector. 
     
     
         7 . (canceled) 
     
     
         8 . 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 according to  claim 4 . 
     
     
         9 . An isolated cell comprising the expression cassette of  claim 4 . 
     
     
         10 . The isolated cell of  claim 9 , wherein the expression cassette is stably integrated into the genome of said cell. 
     
     
         11 - 12 . (canceled) 
     
     
         13 . The isolated nucleic acid molecule of  claim 1 , wherein the nucleic acid sequence is at least 400 base pairs and has at least 95% identity to the nucleic acid sequence of SEQ ID NO:1. 
     
     
         14 . The isolated nucleic acid molecule of  claim 1 , wherein the nucleic acid sequence comprises the nucleic acid sequence of SEQ ID NO:1. 
     
     
         15 . The isolated nucleic acid molecule of  claim 1 , wherein the 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. 
     
     
         16 . The isolated nucleic acid molecule of  claim 15 , wherein the cell is a human cell. 
     
     
         17 . The isolated nucleic acid molecule of  claim 15 , wherein the cell is a rodent cell. 
     
     
         18 . The isolated nucleic acid molecule of  claim 15 , wherein the cell is a Müller cell. 
     
     
         19 . The isolated nucleic acid molecule of  claim 2 , wherein the minimal promoter comprises the nucleic acid sequence of SEQ ID NO:2. 
     
     
         20 . The expression cassette of  claim 4 , wherein the nucleic acid sequence coding for said gene is heterologous to said isolated nucleic acid molecule. 
     
     
         21 . The vector of  claim 5 , wherein said vector is an adeno-associated virus (AAV) vector. 
     
     
         22 . The method of  claim 8 , wherein the isolated cell is a human cell or the cell line or cell population comprises human cells. 
     
     
         23 . The method of  claim 8 , wherein the isolated cell is a rodent cell or the cell line or cell population comprises rodent cells. 
     
     
         24 . The method of  claim 8 , wherein the isolated cell is a Müller cell or the cell line or cell population comprises Müller cells.

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