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Methods and compositions for therapeutic use of RNA interference

A kind of technology of RNA interference, composition

Inactive Publication Date: 2008-09-10
INSERT THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

While overexpression of exogenously introduced transgenes is relatively straightforward in eukaryotic cells, targeted repression of specific genes remains difficult to achieve

Method used

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  • Methods and compositions for therapeutic use of RNA interference
  • Methods and compositions for therapeutic use of RNA interference
  • Methods and compositions for therapeutic use of RNA interference

Examples

Experimental program
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Embodiment

[0277] Having generally described the present invention above, the present invention can be more easily understood with reference to the following examples, which are included to illustrate certain aspects and embodiments of the present invention and not to limit the present invention .

[0278] 1. In vitro delivery of plasmid DNA encoding siRNA

[0279] Human embryonic kidney cells (HEK 293-EcR) were seeded into 6-well culture plates at 200,000 cells per well. These HEK 293-EcR cells have been stably transfected with a plasmid encoding the ecdysone receptor. After 2-3 days, pIND-rev-GFP (a plasmid encoding an inducible element as well as green fluorescent protein) and pTZU6+1 / siRNA (a plasmid encoding the sense and antisense strands of siRNA oligonucleotides) Co-transfect cells. In 0.5ml opti-MEM, the plasmid (see Lee et al. (2002) Nature Biotechnology , 20:500-505) compounded with branched PEI25k-hi-CD polymer (high degree of cyclodextrin grafting). After 4 hours, the ...

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Abstract

The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism, e.g., in a sequence-dependent, PKR-independent manner. In particular, the subject method can be used to alter the growth, survival or differentiation of cells for therapeutic and cosmetic purposes.

Description

[0001] This application is a divisional application of the following application: filing date: November 4, 2002, application number 02826662.5, title of invention "Method and composition for therapeutic use of RNA interference". Background of the invention [0002] The structure and biological behavior of cells are determined by the intracellular gene expression patterns at a specific time. Disturbances in gene expression have long been recognized as the cause of numerous diseases, including various forms of cancer, vascular, neuronal and endocrine disorders. Aberrant expression patterns in the form of amplification, deletion, gene rearrangement, loss or gain of function mutations are known to lead to abnormal behavior of diseased cells. Aberrant gene expression is also thought to be a defense mechanism for some organisms to avoid pathogenic threats. [0003] One of the major challenges in medicine is the regulation of the expression of target genes involved in a wide variety...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00A61K47/40A61K47/42A61K9/14A61P17/16C07H21/02
Inventor M·E·达维斯G·S·詹森S·H·潘
Owner INSERT THERAPEUTICS INC
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