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The emperor's new dystrophin: finding sense in the noise

Trends in molecular medicine, 2015-07, Vol.21 (7), p.417-426

2015

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Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

Human gene therapy, 2007-09, Vol.18 (9), p.798-810

2007

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P.6.6 Splice switching Antisense Oligonucleotides: A “kink” in the design?

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.771-771

2013

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Co-regulation of survival of motor neuron and Bcl-xL expression: Implications for neuroprotection in spinal muscular atrophy

Neuroscience, 2012-09, Vol.220, p.228-236

2012

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Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo

Biochemical Society transactions, 2007-08, Vol.35 (Pt 4), p.826-828

2007

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P.20.2 Restoration of dystrophin expression after skipping of single and double exon DMD duplications in patient-derived cell lines using antisense oligonucleotide and AAV-U7snRNA approaches

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.839-839

2013

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P.11.5 PMO-mediated dystrophin exon 23 skipping restores mitochondrial function in the mdx mouse heart

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.800-800

2013

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Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion

The Journal of cell biology, 2000-03, Vol.148 (5), p.985-995

2000

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G.P.61 Influence of HLA-DRB3 on susceptibility to sporadic inclusion body myositis

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.853-853

2012

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P4.30 Multiple exon skipping strategies to by-pass selected dystrophin mutations

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.713-713

2011

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O10 Development of PMO antisense oligonucleotides for treatment of Spinal muscular atrophy

Neuromuscular disorders : NMD, 2014-03, Vol.24, p.S3-S3

2014

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P.6.5 Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.771-771

2013

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G.P.78 Dystrophin isoforms with incomplete β dystroglycan and syntrophin binding domains retain partial function

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.835-835

2012

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T.I.4 Splice manipulation therapies: Opportunities and challenges

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.831-831

2008

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Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

FEBS letters, 2003-09, Vol.552 (2), p.145-149

2003

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P3.11 Antisense oligomer mediated retention of SMN2 intron and exon 7 leads to a novel SMN transcript and increased functional SMN protein in Spinal Muscular Atrophy fibroblasts

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.685-685

2011

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P18 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies

Neuromuscular disorders : NMD, 2011, Vol.21, p.S11-S11

2011

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D.O.2 Microarray testing for developmental delay reveals an expanded clinical spectrum of dystrophinopathies

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.805-806

2012

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P3.32 Exon/intron 7 inclusion: an alternative mode of splice intervention in spinal muscular atrophy fibroblasts

Neuromuscular disorders : NMD, 2010-10, Vol.20 (9), p.650-651

2010

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T.P.1.06 Induced non-productive splicing to study muscle gene expression

Neuromuscular disorders : NMD, 2009-09, Vol.19 (8), p.578-578

2009

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T.P.2.03 Characterisation of a complex dystrophin mutation: Assume nothing when designing exon skipping strategies

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.757-757

2008

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P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping

Neuromuscular disorders : NMD, 2010, Vol.20, p.S12-S12

2010

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P3.12 Concurrent administration of prednisolone and peptide conjugated PMOs is not contraindicated in the mdx mouse

Neuromuscular disorders : NMD, 2010-10, Vol.20 (9), p.644-644

2010

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T.P.2.01 Antisense oligomer design: Targeting and assay systems

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.756-756

2008

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T.P.2.09 Induced exon skipping in normal and mdx muscle

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.758-759

2008

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The emperor's new dystrophin: finding sense in the noise

Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

P.6.6 Splice switching Antisense Oligonucleotides: A “kink” in the design?

Co-regulation of survival of motor neuron and Bcl-xL expression: Implications for neuroprotection in spinal muscular atrophy

Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo

P.20.2 Restoration of dystrophin expression after skipping of single and double exon DMD duplications in patient-derived cell lines using antisense oligonucleotide and AAV-U7snRNA approaches

P.11.5 PMO-mediated dystrophin exon 23 skipping restores mitochondrial function in the mdx mouse heart

Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion

G.P.61 Influence of HLA-DRB3 on susceptibility to sporadic inclusion body myositis

P4.30 Multiple exon skipping strategies to by-pass selected dystrophin mutations

O10 Development of PMO antisense oligonucleotides for treatment of Spinal muscular atrophy

P.6.5 Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing

G.P.78 Dystrophin isoforms with incomplete β dystroglycan and syntrophin binding domains retain partial function

T.I.4 Splice manipulation therapies: Opportunities and challenges

Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

P3.11 Antisense oligomer mediated retention of SMN2 intron and exon 7 leads to a novel SMN transcript and increased functional SMN protein in Spinal Muscular Atrophy fibroblasts

P18 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies

D.O.2 Microarray testing for developmental delay reveals an expanded clinical spectrum of dystrophinopathies

P3.32 Exon/intron 7 inclusion: an alternative mode of splice intervention in spinal muscular atrophy fibroblasts

T.P.1.06 Induced non-productive splicing to study muscle gene expression

T.P.2.03 Characterisation of a complex dystrophin mutation: Assume nothing when designing exon skipping strategies

P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping

P3.12 Concurrent administration of prednisolone and peptide conjugated PMOs is not contraindicated in the mdx mouse

T.P.2.01 Antisense oligomer design: Targeting and assay systems

T.P.2.09 Induced exon skipping in normal and mdx muscle

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