Dual myostatin and dystrophin exon skipping by morpholino nucleic acid oligomers conjugated to a cell-penetrating peptide is a promising therapeutic strategy for the treatment of duchenne muscular dystrophy

Molecular Therapy Nucleic Acids

Volumn 1, Issue DEC, 2012, Pages e62-

  Malerba, Alberto ^a,b   Kang, Jagjeet K ^a   McClorey, Graham ^c   Saleh, Amer F ^d   Popplewell, Linda ^a   Gait, Michael J ^d   Wood, Matthew J A ^c   Dickson, George ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

^b ROYAL VETERINARY COLLEGE   (United Kingdom)

^c UNIVERSITY OF OXFORD   (United Kingdom)

^d MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Dystrophin; Exon skipping; Myostatin

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CELL PENETRATING PEPTIDE; DYSTROPHIN; HYBRID PROTEIN; MORPHOLINO OLIGONUCLEOTIDE; MYOSTATIN; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER ARGININE RICH CELL PENETRATING PEPTIDE DYSTROPHIN FUSION PROTEIN; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER ARGININE RICH CELL PENETRATING PEPTIDE MYOSTATIN FUSION PROTEIN; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER OCTAGUANIDINE FUSION PROTEIN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; DISEASE MODEL; DRUG CONJUGATION; DRUG EFFECT; DRUG EFFICACY; DRUG TARGETING; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; FEMALE; GENE SILENCING; GENE TARGETING; INFANT; KNOCKOUT GENE; MALE; MOUSE; MUSCLE ATROPHY; MUSCLE HYPERTROPHY; MUSCLE MASS; NONHUMAN; PRIORITY JOURNAL; PROTEIN TARGETING; TIBIALIS ANTERIOR MUSCLE; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

EID: 84887325477     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2012.54     Document Type: Article

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