RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Neuromuscular Disorders
AssigneeProsensa Technologies B.V.
InventorsPeter Christian de Visser, Susan Allegonda Maria Mulders
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Abstract
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.
Claims16
An oligonucleotide comprising a 2′-O-methyl RNA nucleotide residue, having a backbone wherein at least one phosphate moiety is replaced by a phosphorothioate moiety, and comprising one or more 5-methylpyrimidine and/or one or more 2,6-diaminopurine bases, wherein said oligonucleotide is able to hybridize to a repetitive element having as its repetitive nucleotide unit a repetitive nucleotide unit selected from the list consisting of (CAG)n, (GCG)n, (CGG)n, (GAA)n, (GCC)n, (CCG)n, (AUUCU)n, (GGGGCC)n and (CCUG)n.
An oligonucleotide consisting of 2′-O-methyl RNA nucleotide residues, wherein said oligonucleotide comprises a backbone wherein all phosphate moieties are replaced by phosphorothioate moieties, said oligonucleotide further comprising a base sequence consisting of (SEQ ID NO:90).
Dependencies are inferred from the claim text.