Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts

Molecular Therapy

Volumn 20, Issue 11, 2012, Pages 2134-2142

  Cazzella, Valentina ^a   Martone, Julie ^a   Pinnarò, Chiara ^a   Santini, Tiziana ^a   Twayana, Shyam Sundar ^a   Sthandier, Olga ^a   D'amico, Adele ^c   Ricotti, Valeria ^b   Bertini, Enrico ^c   Muntoni, Francesco ^b   Bozzoni, Irene ^a,d  

^a Pasteur Institute   (Italy)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^d SAPIENZA UNIVERSITY OF ROME   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; DYSTROPHIN ASSOCIATED PROTEIN COMPLEX; MICRORNA 1; MICRORNA 29; MICRORNA 29C; NEURONAL NITRIC OXIDE SYNTHASE; SMALL NUCLEOLAR RNA; U1 SMALL NUCLEOLAR RNA; UNCLASSIFIED DRUG;

ADOLESCENT; ARTICLE; BECKER MUSCULAR DYSTROPHY; CELL DIFFERENTIATION; CELL MEMBRANE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHIN GENE; EXON SKIPPING; GENE EXPRESSION; GENE MUTATION; HUMAN; HUMAN CELL; MUSCLE BIOPSY; MYOBLAST; PRESCHOOL CHILD; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; SCHOOL CHILD;

EID: 84869089350     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.178     Document Type: Article

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