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Therapeutic approaches to muscular dystrophy

Human molecular genetics, 2011-04, Vol.20 (R1), p.R69-R78

2011

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Live‐imaging of revertant and therapeutically restored dystrophin in the DmdEGFP‐mdx mouse model for Duchenne muscular dystrophy

Neuropathology and applied neurobiology, 2020-10, Vol.46 (6), p.602-614

2020

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Live-imaging of revertant and therapeutically restored dystrophin in the Dmd EGFP-mdx mouse model for Duchenne muscular dystrophy

Neuropathology and applied neurobiology, 2020-10, Vol.46 (6), p.602-614

2020

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O09 Tricyclo-DNA for the treatment of neuromuscular diseases

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

2014

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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Nature Medicine, 2015-03, Vol.21 (3), p.270-275

2015

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T.O.3 Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.907-907

2012

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P.20.8 AAV genome loss from dystrophic mouse muscles during AAV-U7snRNA-mediated exon skipping therapy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.841-841

2013

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P3.02 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2010-10, Vol.20 (9), p.641-641

2010

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Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

Science (American Association for the Advancement of Science), 2004-12, Vol.306 (5702), p.1796-1799

2004

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P06 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2011, Vol.21, p.S8-S8

2011

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Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies

Gene therapy, 2006-01, Vol.13 (1), p.20-28

2006

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306P Evaluating genetic therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.501

2024

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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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Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides

Nano letters, 2015-07, Vol.15 (7), p.4364-4373

2015

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300P Brain involvement in Duchenne muscular dystrophy (BIND)

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.495

2024

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The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S187-S188

2017

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P.301 - The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S187-S188

2017

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DMD – ANIMAL MODELS: EP.77 Subcellular organization of skeletal muscle dystrophin is restored by tricyclo-DNA mediated exon skipping in the DmdEGFP-mdx mouse model

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S72-S73

2021

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DMD – ANIMAL MODELS

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S72-S73

2021

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In vivo analysis of dystrophin (re-) expression in DmdEGFP and DmdEGFP-mdx reporter mice

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S188

2017

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P.302 - In vivo analysis of dystrophin (re-) expression in DmdEGFP and DmdEGFP-mdx reporter mice

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S188-S188

2017

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Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells

Human gene therapy, 2009-07, Vol.20 (7), p.784-790

2009

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P.286Restoration of dystrophin at critical sites of expression following exon skipping

Neuromuscular disorders : NMD, 2019-10, Vol.29, p.S150-S151

2019

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G.P.3.01 The use of immortalised human fibroblasts from a DMD patient to test exon skipping in vivo

Neuromuscular disorders : NMD, 2007, Vol.17 (9), p.782-782

2007

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Therapeutic approaches to muscular dystrophy

Live‐imaging of revertant and therapeutically restored dystrophin in the DmdEGFP‐mdx mouse model for Duchenne muscular dystrophy

Live-imaging of revertant and therapeutically restored dystrophin in the Dmd EGFP-mdx mouse model for Duchenne muscular dystrophy

O09 Tricyclo-DNA for the treatment of neuromuscular diseases

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

T.O.3 Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD

P.20.8 AAV genome loss from dystrophic mouse muscles during AAV-U7snRNA-mediated exon skipping therapy

P3.02 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

P06 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, November 29th–December 1st 2019

Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies

306P Evaluating genetic therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics

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

Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides

300P Brain involvement in Duchenne muscular dystrophy (BIND)

The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex

P.301 - The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex

DMD – ANIMAL MODELS: EP.77 Subcellular organization of skeletal muscle dystrophin is restored by tricyclo-DNA mediated exon skipping in the DmdEGFP-mdx mouse model

DMD – ANIMAL MODELS

In vivo analysis of dystrophin (re-) expression in DmdEGFP and DmdEGFP-mdx reporter mice

P.302 - In vivo analysis of dystrophin (re-) expression in DmdEGFP and DmdEGFP-mdx reporter mice

Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells

P.286Restoration of dystrophin at critical sites of expression following exon skipping

G.P.3.01 The use of immortalised human fibroblasts from a DMD patient to test exon skipping in vivo

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