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Methods and Compositions for RNAi Mediated Inhibition of Gene Expression in Mammals

Inactive Publication Date: 2020-08-20
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The patent provides methods and compositions for reducing viral replication in mammals using RNAi agents like siRNA or shRNA. These agents can be administered to non-embryonic mammals through a hydrodynamic administration protocol. The patent also provides pharmaceutical preparations for use in research and therapeutic applications. Overall, the patent aims to provide a promising approach for preventing and treating viral infections in mammals.

Problems solved by technology

To date, the Applicants are not aware of any report of successful application of RNAi technology to non-embryonic mammalian organisms.

Method used

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  • Methods and Compositions for RNAi Mediated Inhibition of Gene Expression in Mammals
  • Methods and Compositions for RNAi Mediated Inhibition of Gene Expression in Mammals
  • Methods and Compositions for RNAi Mediated Inhibition of Gene Expression in Mammals

Examples

Experimental program
Comparison scheme
Effect test

experiment a

[0178]control group: RNAs containing the HCV IRES and a luciferase reporter sequence are injected into mice and they glow when this RNA is translated into luciferase protein

Test Group:

[0179]Coinject inhibitor with RNA. Both go to the same cells. Inhibition is expressed as activity (glowing) compared to control group.

experiment b

[0180]Same as experiment A except we inject a DNA that encodes the target RNA along with the inhibitor. The DNA goes to the nucleus of the mouse hepatocytes and is transcribed to give the target RNA. This RNA goes to the cytoplasm of the cells where it interacts with the inhibitor.

[0181]The constructs employed in these experiments are provided in FIG. 4.

The results of these experiments with antisense and DNAzyme inhibitors are provided in FIGS. 5A to 5F.

III. Inhibition of Hepatitis B Virus Replication in Mice by RNA Interference

A. Methods

1. Plasmids

[0182]pTHBV2 (as described in Marion et al., In Frontiers in Viral Hepatitis. (ed. R. F. Schinazi, C. R., and J-P. Sommadossi) 197-209 (Elsevier Science, Amsterdam, 2002)) contains the HBV genome plus a redundancy for the sequences between nucleotides 1067 and 1996 of the HBV genome. HBVU6 RNAi plasmids were cloned using methods described at the website produced by placing http: / / before and “cshl.org:9331 / RNAi / docs / Web_version_of_PCR_stra...

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Abstract

Methods and compositions are provided for modulating, e.g., reducing, coding sequence expression in mammals. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is administered to a non-embryonic mammal, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.

Description

CROSS-REFERENCE TO RELATED APPLICATIONS[0001]This application is a continuation-in-part application of application Ser. No. 10 / 200,002 filed on Jul. 19, 2002; which application (pursuant to 35 U.S.C. § 119 (e)) claims priority to the filing date of the U.S. Provisional Patent Application Ser. No. 60 / 307,411 filed Jul. 23, 2001 and U.S. Provisional Patent Application Ser. No. 60 / 360,664 filed Feb. 27, 2002; the disclosures of which are herein incorporated by reference.INTRODUCTIONField of the Invention[0002]The field of this invention is RNAi.BACKGROUND OF THE INVENTION[0003]Double-stranded RNA induces potent and specific gene silencing through a process referred to as RNA interference (RNAi) or posttranscriptional gene silencing (PTGS). RNAi is mediated by RNA-induced silencing complex (RISC), a sequence-specific, multicomponent nuclease that destroys messenger RNAs homologous to the silencing trigger. RISC is known to contain short RNAs (approximately 22 nucleotides) derived from t...

Claims

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Application Information

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IPC IPC(8): C12N15/113A01K67/027A61K31/70A61K31/7088A61K38/00A61K48/00A61P43/00C07H21/02C12N15/09C12N15/85C12Q1/68
CPCC12N15/1131C12N2310/53Y02A50/30A01K2217/075C12N2310/14A61K38/00A61K31/70A01K67/0275C12N2310/111A61K48/00A61P31/00A61P31/14A61P31/20A61P43/00
Inventor KAY, MARK A.MCCAFFREY, ANTON
Owner THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV
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