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-modified1 - 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.Join the waitlist — get patent alerts
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