# US 9,434,950 B2 · Double-stranded and single-stranded RNA molecules with 5′ Triphosphates and their use for inducing interferon

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- Number: US 9,434,950 B2
- Document ID: US9434950B2
- Status: Granted
- Kind: B2
- Application number: 14/446,324
- Filed: 2014-07-29
- Published: 2015-01-15
- Granted: 2016-09-06
- Earliest claimed: 2005-02-04
- Assignees: City of Hope
- Applicants: City of Hope
- Inventors: John J. Rossi; Dongho Kim; Patric Lundberg; Edouard Cantin
- Web page: https://patentgrep.com/patent/US9434950B2

## CPC

- C12N 15/1131: {against viruses}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- 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 15/117: Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/17: Immunomodulatory nucleic acids
- C12N 2310/18: acting by a non-sequence specific mechanism
- C12N 2310/53: partially self-complementary or closed
- C12N 2320/30: Special therapeutic applications
- C12N 2330/30: chemically synthesised
- C12N 2330/50: Biochemical production, i.e. in a transformed host cell
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

Double-stranded and single-stranded RNA molecules, and their use in methods for inducing interferon are provided. The interferon induction provides anti-viral and other medically useful effects, such as anti-cancer effects. Also provided are methods for reducing or inhibiting interferon induction exhibited by such molecules, particularly siRNA and shRNA molecules produced in vitro.

## Claims

### Claim 1

A method of inducing an anti-cancer response in a mammalian cell, comprising introducing into a mammalian cell an effective amount of a siRNA molecule, wherein the siRNA molecule is double stranded, wherein the siRNA molecule has a 5′-triphosphate on each strand, wherein the siRNA molecule is targeted to a cancer gene, wherein the siRNA molecule induces interferon which mediates an anti-cancer response and wherein the siRNA molecule reduces expression of the targeted cancer gene.

### Claim 2 (depends on 1)

The method of claim 1, wherein each strand of the siRNA molecule is produced in vitro by a phage polymerase.

### Claim 3 (depends on 2)

The method of claim 2, wherein the phage polymerase is T7 RNA polymerase.