# US 7,608,707 B2 · siRNA targeting survivin

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- Number: US 7,608,707 B2
- Document ID: US7608707B2
- Status: Granted
- Kind: B2
- Application number: 11/633,342
- Filed: 2006-12-04
- Published: 2007-03-29
- Granted: 2009-10-27
- Earliest claimed: 2002-11-14
- Assignees: Dharmacon, Inc.
- Inventors: Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Stephen Scaringe; Steven Read
- Web page: https://patentgrep.com/patent/US7608707B2

## CPC

- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- A61P 3/10: for hyperglycaemia, e.g. antidiabetics
- A61P 13/12: of the kidneys
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61P 35/00: Antineoplastic agents
- A61P 35/02: specific for leukemia
- A61P 37/02: Immunomodulators
- C12N 15/1048: {SELEX}
- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- C12N 15/1135: {against oncogenes or tumor suppressor genes}
- C12N 15/1136: {against growth factors, growth regulators, cytokines, lymphokines or hormones}
- C12N 15/1137: {against enzymes (viral enzymes C12N15/1131; receptors C12N15/1138)}
- C12N 15/1138: {against receptors or cell surface proteins}
- C12Y 113/12007: Photinus-luciferin 4-monooxygenase (ATP-hydrolysing) (1.13.12.7), i.e. firefly-luciferase
- C12Y 502/01008: Peptidylprolyl isomerase (5.2.1.8), i.e. cyclophilin
- G16B 20/00: ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B 20/20: Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
- G16B 20/50: Mutagenesis
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2320/10: in screening processes
- C12N 2320/11: for the determination of target sites, i.e. of active nucleic acids
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12Y: ENZYMES
- G16B: BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY

## Abstract

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

## Claims

### Claim 1

An siRNA molecule, wherein said siRNA molecule consists of: (a) a duplex region, wherein said duplex region consists of a sense region and an antisense region, wherein said sense region and said antisense region are each 19-30 bases in length and said antisense region comprises a sequence that is the complement of SEQ ID NO: 451 or SEQ ID NO: 452; and (b) either no overhang regions or at least one overhang region, wherein each overhang region has six or fewer bases.

### Claim 2 (depends on 1)

The siRNA molecule of claim 1, wherein said antisense region and said sense region are each 19-25 nucleotides in length.

### Claim 3 (depends on 2)

The siRNA molecule of claim 2, wherein said antisense region and said sense region are each 19 nucleotides in length.

### Claim 4 (depends on 1)

The siRNA molecule of claim 1, wherein said siRNA molecule has at least one overhang region.

### Claim 5 (depends on 1)

The siRNA molecule of claim 1, wherein said siRNA molecule has no overhang regions.

### Claim 6 (depends on 2)

The siRNA molecule of claim 2, wherein said siRNA molecule has at least one overhang region.

### Claim 7 (depends on 2)

The siRNA molecule of claim 2, wherein said siRNA molecule has no overhang regions.

### Claim 8 (depends on 3)

The siRNA molecule of claim 3, wherein said siRNA molecule has at least one overhang region.

### Claim 9 (depends on 3)

The siRNA molecule of claim 3, wherein said siRNA molecule has no overhang regions.

### Claim 10 (depends on 1)

The siRNA molecule of claim 1, wherein said siRNA molecule comprises a sequence that is the complement of SEQ ID NO: 451.

### Claim 11 (depends on 1)

The siRNA molecule of claim 1, wherein said siRNA molecule comprises a sequence that is the complement of SEQ ID NO: 452.

### Claim 12

A chemically synthesized double stranded siRNA molecule, 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: 451 or SEQ ID NO: 452.

### Claim 13 (depends on 12)

The chemically synthesized double stranded siRNA molecule of claim 12, wherein each strand of said double stranded siRNA molecule is 19-25 nucleotides in length.

### Claim 14 (depends on 12)

The chemically synthesized double stranded siRNA molecule of claim 12, wherein said one strand of said double stranded siRNA molecule comprises a sequence that is the complement of SEQ ID NO: 451.

### Claim 15 (depends on 12)

The chemically synthesized double stranded siRNA molecule of claim 12, wherein said one strand of said double stranded siRNA molecule comprises a sequence that is the complement of SEQ ID NO: 452.

### Claim 16

A pool of at least two siRNAs, wherein said pool comprises a first siRNA and a second siRNA, wherein said first siRNA consists of a duplex region of 19-30 base pairs that has a first antisense region that comprises a sequence that is complementary to SEQ ID NO: 451 and either no overhang regions or at least one overhang region, wherein each overhang region has six or fewer nucleotides; and said second siRNA consists of a duplex region of 19-30 base pairs that has a second antisense region that comprises a sequence that is complementary to SEQ ID NO: 452 and either no overhang regions or at least one overhang region, wherein each overhang region has six or fewer nucleotides.

### Claim 17 (depends on 16)

The pool of claim 16, wherein said first siRNA and said second siRNA each have no overhangs.

### Claim 18 (depends on 16)

The pool of claim 16, wherein said duplex region of said first siRNA and said duplex region of said second siRNA are each 19-25 base pairs in length.

### Claim 19 (depends on 16)

The pool of claim 16, wherein said duplex region of said first siRNA and said duplex region of said second siRNA are each 19 base pairs in length.

### Claim 20 (depends on 13)

The chemically synthesized double-stranded siRNA molecule of claim 13, wherein each strand of said double-stranded siRNA molecule is 19 nucleotides in length.