Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature Medicine

Volumn 12, Issue 2, 2006, Pages 175-177

  Alter, Julia ^a   Lou, Fang ^b   Rabinowitz, Adam ^a   Yin, HaiFang ^a   Rosenfeld, Jeffrey ^c   Wilton, Steve D ^d   Partridge, Terence A ^a   Qi, Long Lu ^c  

^a HAMMERSMITH HOSPITAL   (United Kingdom)

^b UNIVERSITY OF HERTFORDSHIRE   (United Kingdom)

^c CAROLINAS MEDICAL CENTER   (United States)

^d UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MORPHOLINE DERIVATIVE; MORPHOLINO PHOSPHORODIAMIDATE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; FUNCTIONAL PROTEOMICS; GENE FUNCTION; GENE MUTATION; GENETIC TRANSCRIPTION; MOUSE; MOUSE STRAIN; MUSCLE FUNCTION; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SKELETAL MUSCLE; TREATMENT OUTCOME;

ANIMALS; BASE SEQUENCE; DRUG ADMINISTRATION SCHEDULE; DYSTROPHIN; GENE EXPRESSION REGULATION; GENE THERAPY; HUMANS; INJECTIONS, INTRAVENOUS; MALE; MICE; MICE, INBRED C57BL; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGODEOXYRIBONUCLEOTIDES, ANTISENSE;

ANIMALIA;

EID: 32244443828     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm1345     Document Type: Article

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