Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, transgenic mammal, method for inducing RNA interference in an eukaryotic host and application of nucleic acid molecule in the therapy of diseases induced by the expansion of trinucleotide repetitions of CAG type

摘要

The object of the invention is: nucleic acid molecule, express cassette, expression vector, host eukaryotic cell, how to induce RNA interference in the eukaryotic host and how to use nucleic acid molecules in the treatment of diseases caused by the expansion of three nucleotide CAG repetitions. The solution concerns a new concept for the treatment of hereditary neurological diseases in humans caused by the expansion of CAG-type trinucleotide recurrence, using RNA interference technology.