Multiple exon skipping strategies to by-pass dystrophin mutations

Neuromuscular Disorders

Volumn 22, Issue 4, 2012, Pages 297-305

  Adkin, Carl F ^a   Meloni, Penelope L ^a   Fletcher, Susan ^a   Adams, Abbie M ^a   Muntoni, Francesco ^b   Wong, Brenda ^c   Wilton, Steve D ^a  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

Antisense oligomers; Duchenne muscular dystrophy; Dystrophin; Exon skipping; Personalised genetic therapy; Splice switching

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA; MYOD PROTEIN; OLIGOMER;

ARTICLE; CONTROLLED STUDY; DNA ISOLATION; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FIBROBLAST; FRAMESHIFT MUTATION; GENE DELETION; GENE DUPLICATION; GENE MUTATION; GENE SEQUENCE; GENE SWITCHING; HUMAN; HUMAN CELL; HUMAN TISSUE; INTRON; MUSCLE CELL; NUCLEOTIDE SEQUENCE; OPEN READING FRAME; POINT MUTATION; PRIORITY JOURNAL; PROTEIN DOMAIN; PROTEIN MOTIF; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA SEQUENCE; RNA SPLICING; SINGLE NUCLEOTIDE POLYMORPHISM;

CELLS, CULTURED; DYSTROPHIN; EXONS; GENE THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; READING FRAMES; SEQUENCE DELETION;

EID: 84858797261     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2011.10.007     Document Type: Article

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