Systemic delivery of a peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy

Nucleic Acid Therapeutics

Volumn 23, Issue 2, 2013, Pages 109-117

  Leger, Andrew J ^a   Mosquea, Leocadia M ^a   Clayton, Nicholas P ^a   Wu, I Huan ^a   Weeden, Timothy ^a   Nelson, Carol A ^a   Phillips, Lucy ^a   Roberts, Errin ^a   Piepenhagen, Peter A ^a   Cheng, Seng H ^a   Wentworth, Bruce M ^a  

^a SANOFI   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CELL PENETRATING PEPTIDE; MBNL1 PROTEIN; MORPHOLINO OLIGONUCLEOTIDE; RNA BINDING PROTEIN; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONJUGATION; CONTROLLED STUDY; FEMALE; MALE; MOUSE; MYOTONIC DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; RNA SPLICING; TOXICITY;

3' UNTRANSLATED REGIONS; ANIMALS; HUMANS; MICE; MORPHOLINOS; MUTATION; MYOTONIC DYSTROPHY; OLIGONUCLEOTIDES, ANTISENSE; PEPTIDES; PROTEIN-SERINE-THREONINE KINASES; RNA; RNA SPLICING; RNA-BINDING PROTEINS; TRINUCLEOTIDE REPEAT EXPANSION; TRINUCLEOTIDE REPEATS;

MUS;

EID: 84876204431     PISSN: 21593337     EISSN: None     Source Type: Journal    
DOI: 10.1089/nat.2012.0404     Document Type: Article

References (33)

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