RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.
1. A 2′-O-methyl phosphorothioate oligoribonucleotide consisting of the base sequence (XYG) 7 , wherein each X is 5-methyl cytosine and each Y is uracil (SEQ ID NO: 2) or wherein each X is cytosine and each Y is 5-methyluracil (SEQ ID No:3).
2. An oligoribonucleotide according to claim 1 , wherein said oligoribonucleotide is single stranded.
3. A composition comprising an oligoribonucleotide according to claim 1 .
4. A composition according to claim 3 , said composition comprising at least one excipient for enhancing the targeting and/or delivery of said composition to a tissue and/or cell and/or into a tissue and/or cell.
5. A method for treating a subject having a human cis-element repeat instability associated genetic disorder by administering an oligoribonucleotide according to claim 2 to said subject.
6. The method of claim 5 , wherein said subject has the human cis-element repeat instability associated genetic disorder-Huntington Disease.