Post-transcriptional gene silencing by RNAi in mammalian cells
AssigneeThe Trustees of the University of Pennsylvania
InventorAlan Gewirtz
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Abstract
Provided is a method for disrupting cell expression at the mRNA level in mammalian cells using a post-transcriptional gene silencing method known as “RNA mediated interference” or “RNA interference” (“RNAi”). It also provides, for the first time, a demonstration of a RNAi technique that is applicable to human cells and cell lines, as well as for administration to human patients. Thus, this discovery of the value of RNAi for inhibiting mammalian cell expression offers a tool for developing new strategies for blocking gene function, and for producing RNA-based drugs to treat human disease and evaluate vaccine development targets, some of which may not be readily apparent on the basis of sequence information alone.
Claims20
A method for disrupting target cell expression at the mRNA level in a mammalian cell, wherein the method comprises using RNA interference (RNAi) to achieve post-transcriptional gene silencing.
A method for detecting the presence of a target nucleic acid sequence in a biological sample, comprising the steps of:
- transcribing the target sequence into dsRNA,
- exposing the biological sample to the dsRNA, and
- detecting inhibition of gene function of the target nucleic acid sequence in the biological sample, wherein if inhibited, the target nucleic acid sequence is present in the sample.
A method for treating a mammalian subject with an RNA-based disorder or disease by administering to the subject a dsRNA preparation for initiating disruption of target cell expression at the mRNA level, wherein the method comprises using RNAi to achieve post-transcriptional gene silencing.
Dependencies are inferred from the claim text.