Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon

Human Gene Therapy

Volumn 15, Issue 8, 2004, Pages 749-757

  Surono, Agus ^a   Van Khanh, Tran ^a   Takeshima, Yasuhiro ^a   Wada, Hiroko ^b   Yagi, Mariko ^a   Takagi, Miho ^c   Koizumi, Makoto ^c   Matsuo, Masafumi ^a  

^a KOBE UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^b Sakura Ryoikuen Hospital   (Japan)

^c Exploratory Chemistry Research Laboratories   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

2' O METHYLRIBONUCLEIC ACID; 2' O,4' C ETHYLENE BRIDGED NUCLEIC ACID; ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA; NUCLEOTIDE DERIVATIVE; UNCLASSIFIED DRUG;

ARTICLE; CASE REPORT; CHIMERA; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; MALE; MUSCLE FIBER CULTURE; NONSENSE MUTATION; NUCLEIC ACID BASE SUBSTITUTION; PROTEIN EXPRESSION; RNA EDITING; SCHOOL CHILD; WESTERN BLOTTING;

BASE SEQUENCE; BLOTTING, WESTERN; CODON, NONSENSE; DNA PRIMERS; DYSTROPHIN; ETHYLENES; EXONS; GENE THERAPY; HUMANS; IMMUNOHISTOCHEMISTRY; MUSCLE FIBERS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA; RNA EDITING; RNA, MESSENGER; TRANSCRIPTION, GENETIC; TRANSFECTION;

ENA;

EID: 4143075684     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/1043034041648444     Document Type: Article

References (42)

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