Targeted exon skipping to correct exon duplications in the dystrophin gene

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e155-

  Greer, Kane L ^a,b   Lochmüller, Hanns ^c   Flanigan, Kevin ^d   Fletcher, Susan ^a,b   Wilton, Steve D ^a,b  

^a MURDOCH UNIVERSITY   (Australia)

^b UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^c NEWCASTLE UNIVERSITY   (United Kingdom)

^d NATIONWIDE CHILDREN S HOSPITAL   (United States)

Author keywords

Antisense oligomers; Duchenne muscular dystrophy; Duplication mutations; Dystrophin; Exon skipping

Indexed keywords

CELL PENETRATING PEPTIDE; DYSTROPHIN; MESSENGER RNA; MESSENGER RNA PRECURSOR; MORPHOLINO OLIGONUCLEOTIDE; OLIGOMER; OLIGONUCLEOTIDE PHOSPHOROTHIOATE;

ARTICLE; CHICK; CONTROLLED STUDY; DENSITOMETRY; EMBRYO; EXON; EXON DUPLICATION; EXON SKIPPING; GENE DUPLICATION; GENE SEQUENCE; GENETIC CODE; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IN VITRO STUDY; INTRON; MYOBLAST; NONHUMAN; PRIORITY JOURNAL;

EID: 84919761182     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.8     Document Type: Article

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