In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy

Journal of Gene Medicine

Volumn 12, Issue 4, 2010, Pages 354-364

  Wang, Qingsong ^a,b   Yin, HaiFang ^b,c   Camelliti, Patrizia ^b   Betts, Corinne ^b   Moulton, Hong ^d   Lee, Hyunil ^e   Saleh, Amer F ^f   Gait, Michael J ^f   Wood, Matthew J A ^b  

^a CHINA AGRICULTURAL UNIVERSITY   (China)

^b UNIVERSITY OF OXFORD   (United Kingdom)

^c TIANJIN MEDICAL UNIVERSITY   (China)

^d AVI BIOPHARMA INC   (United States)

^e Panagene Incorporated   (South Korea)

^f MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

Author keywords

Antisense oligonucleotide; Duchenne muscular dystrophy; Dystrophin; Exon skipping; In vitro screen

Indexed keywords

2' O METHYLPEPTIDE; ANTISENSE OLIGONUCLEOTIDE; MORPHOLINE DERIVATIVE; PEPTIDE DERIVATIVE; PEPTIDE NUCLEIC ACID; PHOSPHORODIAMIDATE MORPHOLINO; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTISENSE THERAPY; ARTICLE; CHEMICAL STRUCTURE; CLINICAL EVALUATION; CONTROLLED STUDY; DRUG CONJUGATION; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENETIC TRANSFECTION; HEART MUSCLE CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MUSCLE CELL; NONHUMAN; PRIORITY JOURNAL; RISK FACTOR; SCREENING TEST;

ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; BLOTTING, WESTERN; CELLS, CULTURED; DNA PRIMERS; DYSTROPHIN; EXONS; GENE THERAPY; HUMANS; IMMUNOHISTOCHEMISTRY; MICE; MICE, INBRED MDX; MOLECULAR SEQUENCE DATA; MUSCULAR DYSTROPHY, DUCHENNE; MYOCYTES, CARDIAC; OLIGONUCLEOTIDES, ANTISENSE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA PRECURSORS; TRANSCRIPTION, GENETIC; TRANSFECTION;

ANIMALIA;

EID: 77950898446     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1446     Document Type: Article

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