Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Molecular Therapy

Volumn 23, Issue 8, 2015, Pages 1341-1348

  Lu Nguyen, Ngoc B ^a   Jarmin, Susan A ^a   Saleh, Amer F ^b,c   Popplewell, Linda ^a   Gait, Michael J ^b   Dickson, George ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

^b MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

^c ASTRAZENECA   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; B PEPTIDE MORPHOLINO OLIGONUCLEOTIDE CONJUGATE; DYSTROPHIN; MESSENGER RNA PRECURSOR; MORPHOLINO OLIGONUCLEOTIDE; MYOSTATIN; PEPTIDE DERIVATIVE; UNCLASSIFIED DRUG; ARGININE; PEPTIDE;

AGE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; DIAPHRAGM; DRUG MECHANISM; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; MALE; MOUSE; MUSCLE MASS; MUSCLE NECROSIS; NEWBORN; NONHUMAN; OPEN READING FRAME; PROTEIN EXPRESSION; ALTERNATIVE RNA SPLICING; ANIMAL; C57BL MOUSE; CHEMISTRY; DISEASE MODEL; EXON; GENE THERAPY; GENETICS; METABOLISM; NECROSIS; READING FRAME; SKELETAL MUSCLE; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

MUS;

ALTERNATIVE SPLICING; ANIMALS; ANIMALS, NEWBORN; ARGININE; DIAPHRAGM; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; GENETIC THERAPY; MALE; MICE; MICE, INBRED C57BL; MICE, INBRED MDX; MORPHOLINOS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOSTATIN; NECROSIS; OLIGONUCLEOTIDES, ANTISENSE; OPEN READING FRAMES; PEPTIDES; READING FRAMES;

EID: 84938420408     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.88     Document Type: Article

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