Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

摘要

The current invention provides an improved oligonucleotide consisting of a 2’-O-methyl RNA nucleotide residue, having a backbone wherein all phosphate moieties are replaced by phosphorothioate moieties, having a base sequence consisting of (XYG)7 wherein each X is 5-methylcytosine and each Y is Uracil and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.