US2020283771A1PendingUtilityA1

Albumin production and cell proliferation

Assignee: MINA THERAPEUTICS LTDPriority: Jun 21, 2011Filed: May 22, 2020Published: Sep 10, 2020
Est. expiryJun 21, 2031(~4.9 yrs left)· nominal 20-yr term from priority
Inventors:Pål Sætrom
A61P 35/00A61P 1/16C12N 15/113C12N 15/111A61K 31/713C12N 2310/113C12N 2320/30A61K 31/7105Y02A90/10Y02A90/26
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Claims

Abstract

The present invention provides short activating RNA molecules which up-regulate albumin production. The present invention also provides methods of up-regulating albumin production, such methods involving the use of short activating RNA molecules capable of increasing the expression of albumin. The present invention also provides the use of the short activating RNA molecules in therapy, such as treating or preventing a hyperproliferative disorder and/or a disorder characterised by hypoalbuminemia.

Claims

exact text as granted — not AI-modified
1 . A synthetic short activating RNA (saRNA) which up-regulates the expression of a target gene, wherein the target gene is CEBPA. 
     
     
         2 . The saRNA of  claim 1 , wherein the saRNA comprises a strand that has at least 95% sequence identity to a sequence located on the coding strand of the target gene between 500 nucleotides upstream and 500 nucleotides downstream of the transcription start site of the target gene, and
 wherein said strand has 13-30 nucleotides in length.   
     
     
         3 . The saRNA of  claim 1 , wherein the saRNA is double stranded and comprises an anti-sense strand having a sequence of SEQ ID NOs: 14, 16, 18, 20, 22 or 24. 
     
     
         4 . The saRNA of  claim 3 , wherein the saRNA comprises a sense strand having a sequence of SEQ ID NOs: 13, 15, 17, 19, 21 or 23. 
     
     
         5 . The saRNA of  claim 4 , wherein each strand of the saRNA comprises a number of unpaired nucleotides at the 3′ end forming 3′ overhangs. 
     
     
         6 . The saRNA of  claim 5 , wherein said 3′ overhang is UU or UUU. 
     
     
         7 . A method of up-regulating the expression of a target gene in a cell, wherein the target gene is CEBPA, comprising transfecting said cell with the saRNA of  claim 1 . 
     
     
         8 . The method of  claim 7 , wherein the cell is a cancer cell. 
     
     
         9 . The method of  claim 7 , wherein the cell is a liver cell. 
     
     
         10 . The method of  claim 7 , wherein the cell is a liver cancer cell. 
     
     
         11 . A method of treating a hyperproliferative disorder and/or a disorder characterized by hypoalbuminemia in a subject, comprising administering the saRNA of  claim 1  to the subject. 
     
     
         12 . The method of  claim 11 , wherein the subject has liver cancer. 
     
     
         13 . A method of treating liver cirrhosis in a subject, comprising administering the saRNA of  claim 1  to the subject.

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