Gene-based technologies

Case ID: 2016-068-1

Web Published: 12/8/2022

The attractiveness of RNAi for use in therapy lies in its sensitivity and sequence specificity. However, concerns have arisen concerning sequence specificity, e.g. because the wrong strand of the RNA complex may direct the response to the wrong target nucleic acids. Moreover, RNA complexes of a certain size induce a non-specific interferon dependent response, which is also undesirable. This technology describes RNA complexes capable of mediating RNAi comprising an antisense strand and a passenger strand, wherein the strands are 21-23 nucleotides in length and contain at least one non-nucleoside analog to enhance the performance of such RNA complexes, that may be used for therapeutic applications.

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