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filingDate 2012-11-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2021-06-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2a72e3b8eb555ccae9f3490889223ae5
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f1741fd828600c06b6ba5fba76fc8e16
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publicationDate 2021-06-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber ES-2832531-T3
titleOfInvention Oligonucleotides for the treatment of repeat expansion diseases
abstract An antisense oligonucleotide comprising a repeating sequence of three nucleotides having the formula: ** (See formula) ** Nu1, Nu2 and Nu3 are nucleobases selected from the group consisting of adenine, guanine, thymine, uracil, cytosine. and hypoxanthine; n is from about 3 to about 10 which represents the number of repeats of the nucleotide sequence (Nu1, Nu2, Nu3); R1 is a portion of formula ** (See formula) ** q is 0, 1, 2, 3 or 4; R2 is selected from the group consisting of hydrogen, C1-C5 alkyl, and a formamidinyl moiety, and R3 is selected from the group consisting of hydrogen and C1-C5 alkyl, or R2 and R3 join together to form a 5-7 heterocyclic ring members optionally containing an oxygen heteroatom, where the ring may be optionally substituted with a substituent selected from the group consisting of C1-C5 alkyl, phenyl, halogen, and aralkyl; R4 is selected from the group consisting of null, hydrogen, a C1-C6 alkyl, and aralkyl; Rx is selected from the group consisting of -OH, a nucleotide, a cell-penetrating peptide moiety, and piperazinyl; Ry is selected from the group consisting of hydrogen, a C1-C6 alkyl, a nucleotide, a peptide portion, an amino acid, a formamidinyl portion, and acyl; Rz is selected from the group consisting of null, hydrogen, a C1-C6 alkyl, and acyl; and pharmaceutically acceptable salts thereof; wherein the three nucleotide sequence is selected from the group consisting of (CCG), (CTG), (TTC), (NGC), and (GNC); and where N is any nucleotide.
priorityDate 2011-11-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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