Antisense oligonucleotide corrects splice abnormality in hereditary myopathy with lactic acidosis

Gene

Volumn 494, Issue 2, 2012, Pages 231-236

  Sanaker, Petter Schandl ^a,b   Toompuu, Marina ^b,d   McClorey, Graham ^c   Bindoff, Laurence A ^a,b  

^a HAUKELAND UNIVERSITY HOSPITAL   (Norway)

^b UNIVERSITY OF BERGEN   (Norway)

^c UNIVERSITY OF OXFORD   (United Kingdom)

^d TALLINN UNIVERSITY OF TECHNOLOGY   (Estonia)

Author keywords

Antisense oligonucleotide; Cryptic exon; Intronic mutation; Intronic splicing silencer; Metabolic myopathy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CELL PROTEIN; ISCU PROTEIN; MESSENGER RNA; UNCLASSIFIED DRUG;

ADULT; ANTISENSE THERAPY; ARTICLE; CASE REPORT; CONTROLLED STUDY; EXON; FEMALE; FIBROBLAST; GENE MUTATION; GENETIC DISORDER; HEREDITARY MYOPATHY WITH LACTIC ACIDOSIS; HUMAN; HUMAN CELL; INTRON; LACTIC ACIDOSIS; MOLECULARLY TARGETED THERAPY; MYOPATHY; PRIORITY JOURNAL; PSEUDOEXON; RHABDOMYOLYSIS; RNA SPLICING;

ACIDOSIS, LACTIC; ALTERNATIVE SPLICING; CELLS, CULTURED; GENE THERAPY; HUMANS; IRON-SULFUR PROTEINS; MUSCULAR DISEASES; OLIGONUCLEOTIDES, ANTISENSE; TRANSFECTION;

EID: 84856337571     PISSN: 03781119     EISSN: 18790038     Source Type: Journal    
DOI: 10.1016/j.gene.2011.11.021     Document Type: Article

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