Antisense oligonucleotide therapeutics for iron–sulphur cluster deficiency myopathy

[1]  E. Holme,et al.  Clinical manifestation and a new ISCU mutation in iron-sulphur cluster deficiency myopathy. , 2009, Brain : a journal of neurology.

[2]  G. van Ommen,et al.  In vivo comparison of 2′‐O‐methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping , 2009, The journal of gene medicine.

[3]  C. Lázaro,et al.  Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations , 2009, Human mutation.

[4]  T. Rouault,et al.  Iron-sulfur cluster biogenesis and human disease. , 2008, Trends in genetics : TIG.

[5]  M. Holmberg,et al.  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. , 2008, Human molecular genetics.

[6]  D. Hernandez,et al.  Splice mutation in the iron-sulfur cluster scaffold protein ISCU causes myopathy with exercise intolerance. , 2008, American journal of human genetics.

[7]  Johan T den Dunnen,et al.  Local dystrophin restoration with antisense oligonucleotide PRO051. , 2007, The New England journal of medicine.

[8]  M. Ugarte,et al.  Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA. , 2007, American journal of human genetics.

[9]  G. van Ommen,et al.  Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. , 2007, RNA.

[10]  E. Buratti,et al.  RNA structure is a key regulatory element in pathological ATM and CFTR pseudoexon inclusion events , 2007, Nucleic acids research.

[11]  H. Elsässer,et al.  Mechanisms of iron-sulfur protein maturation in mitochondria, cytosol and nucleus of eukaryotes. , 2006, Biochimica et biophysica acta.

[12]  Francisco E. Baralle,et al.  Genomic variants in exons and introns: identifying the splicing spoilers , 2004, Nature Reviews Genetics.

[13]  Michael Zuker,et al.  Mfold web server for nucleic acid folding and hybridization prediction , 2003, Nucleic Acids Res..

[14]  L. Chasin,et al.  Multiple Splicing Defects in an Intronic False Exon , 2000, Molecular and Cellular Biology.

[15]  C. Liew,et al.  A modular domain of NifU, a nitrogen fixation cluster protein, is highly conserved in evolution , 1996, Journal of Molecular Evolution.

[16]  E. Hoffman,et al.  A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. , 2009, Archives of neurology.