Antisense-induced messenger depletion corrects a COL6A2 dominant mutation in ullrich myopathy

Human Gene Therapy

Volumn 23, Issue 12, 2012, Pages 1313-1318

  Gualandi, Francesca ^a   Manzati, Elisa ^a   Sabatelli, Patrizia ^b   Passarelli, Chiara ^a   Bovolenta, Matteo ^a   Pellegrini, Camilla ^a   Perrone, Daniela ^a   Squarzoni, Stefano ^b   Pegoraro, Elena ^c   Bonaldo, Paolo ^c   Ferlini, Alessandra ^a  

^a UNIVERSITY OF FERRARA   (Italy)

^b CNR   (Italy)

^c UNIVERSITY OF PADOVA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

2' O METHYLPHOSPHOROTHIOATE; ANTISENSE OLIGONUCLEOTIDE; COLLAGEN TYPE 6; COLLAGEN TYPE 6A2; UNCLASSIFIED DRUG;

ARTICLE; CONTROLLED STUDY; EXON SKIPPING; EXTRACELLULAR MATRIX; GENE MUTATION; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; MICROFILAMENT; MYOPATHY; SINGLE NUCLEOTIDE POLYMORPHISM;

CELLS, CULTURED; COLLAGEN TYPE VI; EXONS; EXTRACELLULAR MATRIX; FIBROBLASTS; GENES, DOMINANT; HUMANS; MUSCULAR DYSTROPHIES; MUTATION; NONSENSE MEDIATED MRNA DECAY; OLIGORIBONUCLEOTIDES, ANTISENSE; POLYMORPHISM, SINGLE NUCLEOTIDE;

EID: 84871191947     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.109     Document Type: Article

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