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PABPN1 gene therapy for oculopharyngeal muscular dystrophy

Nature communications, 2017-03, Vol.8 (1), p.14848-14848, Article 14848

2017

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Étude transcriptomique spatiale musculaire des myopathies nécrosantes auto-immunes : rôle de l’interféron induit par la nécrose dans le développement des séquelles fibro-adipeuses

La revue de medecine interne, 2023-12, Vol.44, p.A373

2023

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Electrotransfer into skeletal muscle for protein expression

Current gene therapy, 2006-10, Vol.6 (5), p.561-578

2006

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Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

Infection and Immunity, 2009-05, Vol.77 (5), p.2221-2229

2009

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Gene replacement therapy as a novel approach for the treatment of oculopharyngeal muscular dystrophy

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S140-S140

2016

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P9 Adeno-associated virus (AAV)-mediated RNA interference to PABPN1 combined with an optimised resistant transgene for rescue of the muscle specific disease oculopharyngeal muscular dystrophy (OPMD)

Neuromuscular disorders : NMD, 2014-03, Vol.24, p.S9-S9

2014

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G.O.5

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.793-794

2014

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O01 Autologous cell therapy in oculopharyngeal muscular dystrophy (OPMD)

Neuromuscular disorders : NMD, 2014-03, Vol.24, p.S1-S1

2014

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P.16.12 OPMD from the myoblast’s and fibroblast’s point of view

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.826-826

2013

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P176 Immortalized human muscle cells: easy-to-use models to study neuromuscular diseases

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S109-S109

2023

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268P Age and sex affect human skeletal muscle secretome

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.77

2024

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594P FUS/TLS as a potential regulator of PABPN1 in skeletal muscle?

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.349

2024

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Saving dopaminergic neurons from endoplasmic reticulum (ER) stress - A breakthrough approach to fight Parkinson's disease

Parkinsonism & related disorders, 2024-05, Vol.122, p.106823, Article 106823

2024

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G.P.106 Immunohistochemical study of cricopharyngeal muscle in oculopharyngeal muscular dystrophy

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.900-900

2012

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P166 Deciphering the genetic cause of oculopharyngodistal myopathy in a French cohort using Cas9-targeted long-read sequencing

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S141-S141

2023

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Optical imaging of luminescence for in vivo quantification of gene electrotransfer in mouse muscle and knee

BMC biotechnology, 2006-03, Vol.6 (1), p.16-16

2006

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P2.45 Proteomic studies of muscle progenitor cells in affected and non-affected oculopharyngeal muscular dystrophy muscles

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.674-674

2011

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Editorial: Skeletal Muscle Immunometabolism

Frontiers in physiology, 2021-04, Vol.12, p.683088-683088

2021

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A negative feedback loop between fibroadipogenic progenitors and muscle fibres involving endothelin promotes human muscle fibrosis

Journal of cachexia, sarcopenia and muscle, 2022-06, Vol.13 (3), p.1771-1784

2022

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Invited review: Stem cells and muscle diseases: advances in cell therapy strategies

Neuropathology and applied neurobiology, 2015-04, Vol.41 (3), p.270-287

2015

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D.P.4.11 Expression and siRNA targeting of PABPN1 as a model for oculopharyngeal muscular dystrophy (OPMD)

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.799-799

2008

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G.P.8.08 Codon optimisation of microdystrophin results in improvements in expression and physiological outcome in the mdx mouse following AAV8 gene transfer

Neuromuscular disorders : NMD, 2008-10, Vol.18 (9), p.784-784

2008

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FSHD / OPMD / MYOTONIC DYSTROPHY: P.240 Targeting unfolded protein response to resolve aggresomes accumulation in oculopharyngeal muscular dystrophy

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S116-S116

2020

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FSHD / OPMD / MYOTONIC DYSTROPHY

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S116

2020

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Fibroadipogenic Progenitors Regulate the Basal Proliferation of Satellite Cells and Homeostasis of Pharyngeal Muscles via HGF Secretion

Frontiers in cell and developmental biology, 2022-05, Vol.10, p.875209-875209

2022

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PABPN1 gene therapy for oculopharyngeal muscular dystrophy

Étude transcriptomique spatiale musculaire des myopathies nécrosantes auto-immunes : rôle de l’interféron induit par la nécrose dans le développement des séquelles fibro-adipeuses

Electrotransfer into skeletal muscle for protein expression

Generation of High-Titer Neutralizing Antibodies against Botulinum Toxins A, B, and E by DNA Electrotransfer

Gene replacement therapy as a novel approach for the treatment of oculopharyngeal muscular dystrophy

P9 Adeno-associated virus (AAV)-mediated RNA interference to PABPN1 combined with an optimised resistant transgene for rescue of the muscle specific disease oculopharyngeal muscular dystrophy (OPMD)

G.O.5

O01 Autologous cell therapy in oculopharyngeal muscular dystrophy (OPMD)

P.16.12 OPMD from the myoblast’s and fibroblast’s point of view

P176 Immortalized human muscle cells: easy-to-use models to study neuromuscular diseases

268P Age and sex affect human skeletal muscle secretome

594P FUS/TLS as a potential regulator of PABPN1 in skeletal muscle?

Saving dopaminergic neurons from endoplasmic reticulum (ER) stress - A breakthrough approach to fight Parkinson's disease

G.P.106 Immunohistochemical study of cricopharyngeal muscle in oculopharyngeal muscular dystrophy

P166 Deciphering the genetic cause of oculopharyngodistal myopathy in a French cohort using Cas9-targeted long-read sequencing

Optical imaging of luminescence for in vivo quantification of gene electrotransfer in mouse muscle and knee

P2.45 Proteomic studies of muscle progenitor cells in affected and non-affected oculopharyngeal muscular dystrophy muscles

Editorial: Skeletal Muscle Immunometabolism

A negative feedback loop between fibroadipogenic progenitors and muscle fibres involving endothelin promotes human muscle fibrosis

Invited review: Stem cells and muscle diseases: advances in cell therapy strategies

D.P.4.11 Expression and siRNA targeting of PABPN1 as a model for oculopharyngeal muscular dystrophy (OPMD)

G.P.8.08 Codon optimisation of microdystrophin results in improvements in expression and physiological outcome in the mdx mouse following AAV8 gene transfer

FSHD / OPMD / MYOTONIC DYSTROPHY: P.240 Targeting unfolded protein response to resolve aggresomes accumulation in oculopharyngeal muscular dystrophy

FSHD / OPMD / MYOTONIC DYSTROPHY

Fibroadipogenic Progenitors Regulate the Basal Proliferation of Satellite Cells and Homeostasis of Pharyngeal Muscles via HGF Secretion

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