Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats
AssigneeInstytut Chemii Bioorganicznej Pan
InventorsWlodzimierz Krzyzosiak, Marta Olejniczak, Paulina Galka-Marciniak, Agnieszka Fiszer
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Abstract
Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.
Claims14
A nucleic acid molecule composed of a duplex and loop, in which one of the duplex strands, the guide strand, comprises a sequence chosen from SEQ ID NO:
- 1 and SEQ ID NO:
- 2, and the other strand of the duplex, the passenger strand, is at least 80% complementary to the guide strand, wherein the nucleic acid molecule forms a hairpin structure in a cell.
Dependencies are inferred from the claim text.