Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells

Proceedings of the National Academy of Sciences of the United States of America

Volumn 99, Issue 14, 2002, Pages 9456-9461

  De Angelis, Fernanda Gabriella ^a   Sthandier, Olga ^a   Berarducci, Barbara ^b   Toso, Silvia ^a   Galluzzi, Giuliana ^c   Ricci, Enzo ^c   Cossu, Giulio ^a,b   Bozzoni, Irene ^a  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c CATHOLIC UNIVERSITY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA PRECURSOR; SMALL NUCLEAR RNA;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; AMINO ACID SEQUENCE; ANIMAL CELL; ARTICLE; CHIMERA; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE ACTIVITY; GENE CONSTRUCT; GENE DELETION; GENE MUTATION; NONHUMAN; OPEN READING FRAME; PRIORITY JOURNAL; PROTEIN SYNTHESIS; RNA SPLICING;

ANIMALS; DYSTROPHIN; EXONS; FEMALE; GENE THERAPY; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OOCYTES; RNA; RNA PRECURSORS; RNA SPLICING; RNA, ANTISENSE; RNA, SMALL NUCLEAR; SEQUENCE DELETION; TRANSDUCTION, GENETIC; XENOPUS LAEVIS;

ANIMALIA;

EID: 0037047111     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.142302299     Document Type: Article

References (38)