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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

Human molecular genetics, 2012-04, Vol.21 (7), p.1625-1638

2012

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MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells

Cancer research (Chicago, Ill.), 2013, Vol.73 (1), p.341-352

2013

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Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

Molecular therapy. Nucleic acids, 2017-12, Vol.9 (C), p.155-161

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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Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal

PloS one, 2013-08, Vol.8 (8), p.e72147-e72147

2013

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Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency

Frontiers in medicine, 2022-02, Vol.9, p.827416-827416

2022

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A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice

Human gene therapy, 2013-03, Vol.24 (3), p.331

2013

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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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Smart functional nucleic acid chimeras: Enabling tissue specific RNA targeting therapy

RNA biology, 2015, Vol.12 (4), p.412-425

2015

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Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse

Molecular therapy, 2007-09, Vol.15 (9), p.1587-1592

2007

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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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Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the mdx Mouse

Proceedings of the National Academy of Sciences - PNAS, 2001-01, Vol.98 (1), p.42-47

2001

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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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Molecular analysis of a spontaneous dystrophin `knockout' dog

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

1999

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A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

Human gene therapy, 2013-03, Vol.24 (3), p.331-342

2013

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Redirecting splicing to address dystrophin mutations: molecular by-pass surgery

Progress in molecular and subcellular biology, 2006, Vol.44, p.161

2006

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Antisense suppression of donor splice site mutations in the dystrophin gene transcript

Molecular genetics & genomic medicine, 2013-09, Vol.1 (3), p.162-173

2013

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Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes

Health policy and technology, 2014-12, Vol.3 (4), p.325-335

2014

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Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis

Journal of applied physiology (1985), 2008-08, Vol.105 (2), p.662-668

2008

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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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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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Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

Genetic vaccines and therapy, 2006-05, Vol.4 (1), p.3-3, Article 3

2006

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Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?

Neuromuscular disorders : NMD, 1997-07, Vol.7 (5), p.329-335

1997

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Outcomes of cardiac resynchronization therapy in patients with versus those without atrial fibrillation: A systematic review and meta-analysis

Heart rhythm, 2011-07, Vol.8 (7), p.1088-1094

2011

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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells

Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

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

Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal

Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice

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

Smart functional nucleic acid chimeras: Enabling tissue specific RNA targeting therapy

Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse

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

Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the mdx Mouse

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

Molecular analysis of a spontaneous dystrophin `knockout' dog

A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

Redirecting splicing to address dystrophin mutations: molecular by-pass surgery

Antisense suppression of donor splice site mutations in the dystrophin gene transcript

Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes

Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis

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

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

Terminal antisense oligonucleotide modifications can enhance induced exon skipping

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?

Outcomes of cardiac resynchronization therapy in patients with versus those without atrial fibrillation: A systematic review and meta-analysis

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