Evaluation of 2′-Deoxy-2′-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

Molecular Therapy Nucleic Acids

Volumn 4, Issue , 2015, Pages e265-

  Jirka, Silvana M G ^a   Tanganyika De Winter, Christa L ^a   Boertje Van Der Meulen, Joke W ^a   Van Putten, Maaike ^a   Hiller, Monika ^a   Vermue, Rick ^b   De Visser, Peter C ^b   Aartsma Rus, Annemieke ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

2 deoxy 2 fluoro; 2 O methyl phosphorothioate; antisense oligonucleotide; dystrophin; exon skipping

Indexed keywords

2' DEOXY 2' FLUORO ANTISENSE OLIGONUCLEOTIDE DERIVATIVE; ANTISENSE OLIGONUCLEOTIDE; MESSENGER RNA PRECURSOR; PHOSPHOROTHIOIC ACID; PHOSPHOROTHIOIC ACID DERIVATIVE; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHEMICAL MODIFICATION; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; HUMAN; HUMAN CELL; HUMAN CELL CULTURE; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA SPLICING;

EID: 84948991145     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2015.39     Document Type: Article

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