Antisense oligonucleotide therapeutics for iron–sulphur cluster deficiency myopathy
Author:
Publisher
Elsevier BV
Subject
Genetics(clinical),Clinical Neurology,Neurology,Pediatrics, Perinatology, and Child Health
Reference16 articles.
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2. Myopathy with lactic acidosis is linked to chromosome 12q23.3–24.11 and caused by an intron mutation in the ISCU gene resulting in a splicing defect;Olsson;Hum Mol Genet,2008
3. Clinical manifestation and a new ISCU mutation in iron–sulphur cluster deficiency myopathy;Kollberg;Brain,2009
4. Multiple splicing defects in an intronic false exon;Sun;Mol Cell Biol,2000
5. RNA structure is a key regulatory element in pathological ATM and CFTR pseudoexon inclusion events;Buratti;Nucleic Acids Res,2007
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