Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice

Human Gene Therapy

Volumn 17, Issue 5, 2006, Pages 565-574

  Denti, Michela Alessandra ^a   Rosa, Alessandro ^a   D'Antona, Giuseppe ^b   Sthandier, Olga ^a   De Angelis, Fernanda Gabriella ^a   Nicoletti, Carmine ^a   Allocca, Mariacarmela ^c   Pansarasa, Orietta ^b   Parente, Valeria ^b   Musarò, Antonio ^a   Auricchio, Alberto ^c   Bottinelli, Roberto ^b   Bozzoni, Irene ^a  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

^b UNIVERSITY OF PAVIA   (Italy)

^c TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY RNA; DYSTROPHIN; MESSENGER RNA; SMALL NUCLEAR RIBONUCLEOPROTEIN; SMALL NUCLEAR RNA;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHIMERA; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE EXPRESSION; MOUSE; MOUSE STRAIN; MUSCLE FUNCTION; NONHUMAN; OPEN READING FRAME; POINT MUTATION; PROTEIN SYNTHESIS; RNA VECTOR; SPLICEOSOME; STOP CODON; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BASE SEQUENCE; DEPENDOVIRUS; DNA, RECOMBINANT; DYSTROPHIN; GENE THERAPY; GENETIC VECTORS; INJECTIONS, INTRAMUSCULAR; MICE; MICE, INBRED MDX; MOLECULAR SEQUENCE DATA; MUSCLE FIBERS; MUSCULAR DYSTROPHY, ANIMAL; RECOMBINANT FUSION PROTEINS; RNA, ANTISENSE; RNA, SMALL NUCLEAR;

ANIMALIA;

EID: 33646930313     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2006.17.565     Document Type: Article

References (29)

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