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The sequence of siRNA, vector, molecular target for siRNA reagents and vectors introduced to cells and tissues, the manner of assessment of specifity of silencing of a mutated transcript, the manner of testing of influences of RNA interference route of enz
The sequence of siRNA, vector, molecular target for siRNA reagents and vectors introduced to cells and tissues, the manner of assessment of specifity of silencing of a mutated transcript, the manner of testing of influences of RNA interference route of enz
The subject of the present invention are a siRNA sequence, a vector, a molecular target for siRNA reagents and vectors introduced into cells and tissues, a method of evaluating the specificity of the silencing of the mutated transcript, a method of examining the interactions of the RNA interference pathway with transcripts containing repeating sequences as well as the application of a siRNA sequence and the vector in the therapy of trinucleotide repeat expansion diseases (TREDs). The solution relates to a new concept in the treatment of inherited neurological diseases caused by the expansion of tri-nucleotide elements using RNA interference and new tools for the embodiment of said concept.
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