Method for Obtaining an Enriched Population of siRNA-Expressing Cells Useful in Gene Therapy

The invention relates to a method of enriching a population of mammalian cells having an RNA interference (RNAi) sequence by providing eukaryotic (e.g., mammalian) cells containing a target gene. The invention defines a unique plasmid vector able to enrich siRNA-expressing cells to a high level of purity using magnetic bead sorting. This invention brings an immediate improvement in the efficiency of delivery of any siRNA.

This invention circumvents the limitations of transfection efficiency while retaining desirable sustained RNAi expression. By introducing at least one nucleic acid capable of expressing an RNAi molecule and a separation marker, the method obtains cells capable of expressing inhibitory RNA. The invention can be used to target cellular genes, exogenous genes, viral genes, and transgenes for RNA inhibition. In one embodiment, the invention relates to transfection of mammalian cells and retains desirable sustained RNAi expression.

The ability to specifically downregulate gene expression is a powerful means of gene function analysis in many model systems. Until recently, the only available method of targeted gene downregulation has been expensive and cumbersome gene-knockout technology in mice. Only recently, the characterization of RNAi in C. elegans and plants led to the discovery of a similar system in mammalian cells. Mammalian cells, however, lack RNA-dependent polymerase that are present in C. elegans and plants and facilitate amplification and sustained expression of the interfering RNA signal.

Stage of Development
PCT application has been filed with the USPTO under publication number US 2006/0216823 A1. It is available for further developmental research support and licensing under either exclusive or non-exclusive terms.

Additional Info

Inventor(s): Vicente Planelles, Erik Zimmerman

Type of Offer: Licensing

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