Nucleic acid molecule and method of targeting gene expression to gliomas

Abstract

There is presently provided a nucleic acid molecule comprising a glial-specific promoter; a coding sequence for a transgene; and a plurality of miRNA target sites. Each miRNA target site binds a miRNA that is down-regulated in a glioma cell compared to a normal glial cell, and the glial-specific promoter and the plurality of miRNA target sites are both operably linked to the coding sequence for the transgene.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of and priority to US Provisional Patent Application No. 61 / 071,814, filed May 19, 2008, the contents of which are incorporated herein by reference in their entirety. technical field [0003] The present invention generally relates to nucleic acid molecules, expression vectors and methods for targeting gene expression in glioma. Background technique [0004] Specific gene expression in a selected cell or tissue type can be achieved by targeted gene delivery using a delivery vehicle that binds a ligand through which it binds to a cell surface receptor unique to the target cell. Specific gene expression can also be achieved through targeted transcription using cell-specific promoters and enhancers. Cell-specific promoters are one of the main ways to restrict specific cellular functions to specific differentiated cell types. The ability of these promoters to direct the transcription o...

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Problems solved by technology

However, since the natural infectious spectrum of most viral vectors is not restricted to tumor tissues, the potential leakage of intratumorally injected vectors into normal tissues is a concern and may cause collateral damage to healthy cells

Although this disruption is tolerable in some organs, it can have serious consequences in sensitive organs such as the brain

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