US2010004142A1PendingUtilityA1

siRNA targeting myeloid cell Leukemia sequence 1

Assignee: DHARMACON INCPriority: Nov 14, 2002Filed: Aug 12, 2009Published: Jan 7, 2010
Est. expiryNov 14, 2022(expired)· nominal 20-yr term from priority
A61P 35/02A61P 3/10A61P 37/02A61P 35/00A61P 25/28C12N 15/1136C12N 2310/14G16B 20/00C12N 15/113C12N 15/1138A61K 31/713C12N 2320/11A61P 21/00C12Y 502/01008A61P 13/12C12N 15/1135C12N 15/1048C12N 2320/10C12N 15/1137C12Y 113/12007C12N 15/111G16B 20/20G16B 20/50
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Claims

Abstract

Efficient sequence specific gene silencing of myeloid cell leukemia sequence 1 is possible through the use of siRNA technology. By selecting particular siRNAs directed to myeloid cell leukemia sequence 1 by rationale design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes.

Claims

exact text as granted — not AI-modified
1 . An siRNA molecule consisting of: (a) a sense region and an antisense region, wherein said sense region and said antisense region together form a duplex region consisting of 19-30 base pairs and said antisense region comprises a sequence that is the complement of a sequence selected from SEQ ID NO 439-460 and 462-463; and (b) zero to four overhang regions, wherein each overhang region is six or fewer nucleotides in length. 
     
     
         2 . The siRNA molecule of  claim 1 , wherein said antisense region and said sense region are each 19-25 nucleotides in length. 
     
     
         3 . The siRNA molecule of  claim 2 , wherein said antisense region and said sense region are each 19 nucleotides in length. 
     
     
         4 . The siRNA molecule of  claim 1 , wherein said siRNA molecule has at least one overhang region. 
     
     
         5 . The siRNA molecule of  claim 1 , wherein said siRNA molecule has only one overhang region. 
     
     
         6 . The siRNA molecule of  claim 1 , wherein said siRNA molecule has two overhang regions. 
     
     
         7 . The siRNA molecule of  claim 1 , wherein said siRNA molecule has no overhang regions. 
     
     
         8 . The siRNA molecule of  claim 2 , wherein said siRNA molecule has at least one overhang region. 
     
     
         9 . The siRNA molecule of  claim 2 , wherein said siRNA molecule has no overhang regions. 
     
     
         10 . The siRNA molecule of  claim 3 , wherein said siRNA molecule has at least one overhang region. 
     
     
         11 . The siRNA molecule of  claim 3 , wherein said siRNA molecule has no overhang regions. 
     
     
         12 . A chemically synthesized double stranded siRNA molecule that directs cleavage of an mRNA via RNA interference, wherein:
 a. each strand of said double stranded siRNA molecule is between 19 and 30 nucleotides in length; and   b. one strand of said double stranded siRNA molecule comprises a sequence that is the complement of SEQ ID NO 464.   
     
     
         13 . The chemically synthesized double stranded siRNA molecule of  claim 12 , wherein each strand of said double stranded siRNA molecule is 19 nucleotides in length. 
     
     
         14 . An siRNA molecule, wherein said siRNA molecule consists of: (a) a sense region, wherein said sense region consists of a sequence that is selected from SEQ ID NO 439-460 and 462-463, and an antisense region, wherein said antisense region consists of a sequence that is the complement the sequence that is selected; and (b) zero to four overhang regions, wherein each overhang region is six or fewer nucleotides in length. 
     
     
         15 . The siRNA molecule of  claim 14 , wherein said siRNA molecule has no overhang regions. 
     
     
         16 . The siRNA molecule of  claim 14 , wherein said siRNA molecule has one overhang region. 
     
     
         17 . The siRNA molecule of  claim 14 , wherein said siRNA molecule has two overhang regions. 
     
     
         18 . A pool of siRNA molecules, wherein said pool comprises a first siRNA molecule, wherein said first siRNA molecule is the siRNA molecule of  claim 1  and a second siRNA molecule, wherein the second siRNA molecule consists of: (a) a sense region and an antisense region, wherein said sense region and said antisense region of said second siRNA molecule together form a duplex region consisting of 19-30 base pairs and said antisense region of said second siRNA molecule comprises a sequence that is the complement of a sequence selected from the group of sequences consisting of SEQ ID NO: 439-460 and 462-463; and (b) zero to four overhang regions, wherein each overhang region is six or fewer nucleotides in length. 
     
     
         19 . The pool of  claim 18 , wherein the antisense region of said second siRNA molecule comprises a sequence that is the complement of SEQ ID NO: 461. 
     
     
         20 . A pool of siRNA molecules, wherein said pool comprises a first siRNA molecule, wherein said first siRNA molecule is the siRNA molecule of  claim 18  and a second siRNA molecule, wherein the second siRNA molecule consists of: (a) a sense region and an antisense region, wherein said sense region and said antisense region of said second siRNA molecule together form a duplex region consisting of 19 base pairs and said antisense region of said second siRNA molecule has a sequence that is the complement of SEQ ID NO: 461; and (b) zero to four overhang regions, wherein each overhang region is six or fewer nucleotides in length.

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