THERAPEUTIC MOLECULES FOR USE IN THE SUPPRESSION OF PARKINSON'S DISEASE

摘要

There is provided a double stranded nucleic acid (dsNA) molecule comprising a nucleic acid sense strand and an RNA antisense strand wherein the RNA antisense strand binds to position 6176 on a target RNA nucleotide sequence that comprises the nucleotide sequence of SEQ ID NO: 10; wherein at least a portion of the RNA antisense strand and the nucleic acid sense strand together define a base-paired nucleic acid duplex wherein the nucleotides of the antisense RNA strand define consecutively numbered antisense nucleotide positions, said numbers increasing in a 5' to 3' direction on the antisense strand, with position 1 (p1 ) defined as the extreme 5' nucleotide present on the RNA antisense strand of the nucleic acid duplex that is base paired with a corresponding nucleotide present on the nucleic acid sense strand of the nucleic acid duplex; and wherein the RNA antisense strand has a uracil nucleotide located at any one of positions p1 to p9 that binds to an adenine nucleotide located at position 6176 of an RNA nucleotide sequence comprising the nucleotide sequence of SEQ ID NO: 10. Also provided are vectors encoding said dsNA, and therapeutic uses of the dsNA molecule for suppressing/preventing Parkinson's disease.