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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature medicine, 2006-02, Vol.12 (2), p.175-177

2006

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Translational development of splice-modifying antisense oligomers

Expert opinion on biological therapy, 2017-01, Vol.17 (1), p.15-30

2017

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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

Nature medicine, 2003-08, Vol.9 (8), p.1009-1014

2003

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Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle

Human molecular genetics, 2003-08, Vol.12 (15), p.1801-1811

2003

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Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

The journal of gene medicine, 2002-11, Vol.4 (6), p.644-654

2002

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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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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides

Neuromuscular disorders : NMD, 1999-07, Vol.9 (5), p.330-338

1999

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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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The Regrowth of Axons within Tissue Defects in the CNS Is Promoted by Implanted Hydrogel Matrices That Contain BDNF and CNTF Producing Fibroblasts

Experimental neurology, 2001-07, Vol.170 (1), p.72-84

2001

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RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes

Current gene therapy, 2005-10, Vol.5 (5), p.467-483

2005

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A mutation in the α tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy

Nature genetics, 1995-01, Vol.9 (1), p.75-79

1995

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Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?

Neuromuscular disorders : NMD, 2005-06, Vol.15 (6), p.399-402

2005

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Multiple QTLs influencing triglyceride and HDL and total cholesterol levels identified in families with atherogenic dyslipidemia

Journal of lipid research, 2005-10, Vol.46 (10), p.2202-2213

2005

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Homozygosity for a nonsense mutation in the alpha-tropomyosin slow gene TPM3 in a patient with severe infantile nemaline myopathy

Neuromuscular disorders : NMD, 1999-12, Vol.9 (8), p.573-579

1999

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Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene

The journal of gene medicine, 2003-06, Vol.5 (6), p.518-527

2003

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Terminal antisense oligonucleotide modifications can enhance induced exon skipping

Neuromuscular disorders : NMD, 2005-10, Vol.15 (9), p.622-629

2005

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Molecular analysis of a spontaneous dystrophin `knockout' dog

Neuromuscular disorders : NMD, 1999-07, Vol.9 (5), p.289-295

1999

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Autosomal dominant distal myopathy : linkage to chromosome 14

American journal of human genetics, 1995-02, Vol.56 (2), p.422-427

1995

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Novel mutation in the myelin protein zero gene in a family with intermediate hereditary motor and sensory neuropathy

Journal of neurology, neurosurgery and psychiatry, 1999-08, Vol.67 (2), p.174-179

1999

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Use of polymerase chain reaction for rapid diagnosis of tuberculosis

Journal of Clinical Microbiology, 1992-01, Vol.30 (1), p.255-258

1992

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Bandstab: A PCR-Based Alternative to Cloning PCR Products

BioTechniques, 1997-04, Vol.22 (4), p.642-645

1997

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Dystrophin gene transcripts skipping the mdx mutation

Muscle & nerve, 1997-06, Vol.20 (6), p.728-734

1997

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A splice-site mutation causing ovine McArdle's disease

Neuromuscular disorders : NMD, 1997-07, Vol.7 (5), p.336-342

1997

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Gene therapy and molecular approaches to the treatment of hereditary muscular disorders

Current opinion in neurology, 2000-10, Vol.13 (5), p.553-560

2000

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Apolipoprotein-E genotyping in diagnosis of Alzheimer's disease

The Lancet (British edition), 1996-08, Vol.348 (9025), p.483-483

1996

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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Translational development of splice-modifying antisense oligomers

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle

Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

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

Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides

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

The Regrowth of Axons within Tissue Defects in the CNS Is Promoted by Implanted Hydrogel Matrices That Contain BDNF and CNTF Producing Fibroblasts

RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes

A mutation in the α tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy

Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?

Multiple QTLs influencing triglyceride and HDL and total cholesterol levels identified in families with atherogenic dyslipidemia

Homozygosity for a nonsense mutation in the alpha-tropomyosin slow gene TPM3 in a patient with severe infantile nemaline myopathy

Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene

Terminal antisense oligonucleotide modifications can enhance induced exon skipping

Molecular analysis of a spontaneous dystrophin `knockout' dog

Autosomal dominant distal myopathy : linkage to chromosome 14

Novel mutation in the myelin protein zero gene in a family with intermediate hereditary motor and sensory neuropathy

Use of polymerase chain reaction for rapid diagnosis of tuberculosis

Bandstab: A PCR-Based Alternative to Cloning PCR Products

Dystrophin gene transcripts skipping the mdx mutation

A splice-site mutation causing ovine McArdle's disease

Gene therapy and molecular approaches to the treatment of hereditary muscular disorders

Apolipoprotein-E genotyping in diagnosis of Alzheimer's disease

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