Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2′-deoxyuridine nucleotides induce exon-skipping in vitro
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DOI: 10.1039/c7ra10964d
Abstract: Chemically-modified antisense oligonucleotide-mediated exon-skipping has been validated as a therapeutic strategy for tackling several disease pathologies, particularly duchenne muscular dystrophy. To date, only sugar-modified and internucleotide linkage-modified oligonucleotide chemistries have been explored for exon-skipping applications.… read more here.
Keywords: nucleobase modified; antisense oligonucleotides; modified antisense; phenyltriazol deoxyuridine ... See more keywords