A SIRNA SEQUENCE, 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 ENZYMES ON THE

摘要

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.