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PEPTIDE-LINKED MORPHOLINO ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF MYOTONIC DYSTROPHY.
PEPTIDE-LINKED MORPHOLINO ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF MYOTONIC DYSTROPHY.
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机译:肽键连接的吗啉类反义寡核苷酸用于治疗肌强直性营养不良。
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摘要
Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3' untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).
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