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Intracellular Trafficking of Adeno-Associated Virus Vectors: Routing to the Late Endosomal Compartment and Proteasome Degradation

Journal of Virology, 2001-02, Vol.75 (4), p.1824-1833

2001

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Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S209-S209

2016

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G.P.43

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.807-808

2014

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G.P.41

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.807-807

2014

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Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S274-S274

2015

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P.4.3 Intravenous infusion of AAV8–MTM1 prolongs life and ameliorates severe muscle pathology in mouse and dog models of X-linked myotubular myopathy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.760-760

2013

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AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy

Molecular therapy, 2020-02, Vol.28 (2), p.382-393

2020

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P1.44 Neuromuscular junction abnormalities and myotubular myopathy

Neuromuscular disorders : NMD, 2010-10, Vol.20 (9), p.613-614

2010

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Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice

Proceedings of the National Academy of Sciences - PNAS, 2016-12, Vol.113 (50), p.14432-14437

2016

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T.O.4 Development of AAV-gene and protein-based therapies for X-linked myotubular myopathy

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

2012

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CONGENITAL MYOPATHIES – NEMALINE MYOPATHIES: EP.40 Neuromuscular junction defects in ACTA1-related nemaline myopathy

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S61-S62

2021

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CONGENITAL MYOPATHIES – NEMALINE MYOPATHIES

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S61-S62

2021

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O.32Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in muscle of DM1 mice

Neuromuscular disorders : NMD, 2019-10, Vol.29, p.S125-S125

2019

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CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S181

2017

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P.278 - CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S181-S181

2017

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Iodine release from oxidised Zircaloy cladding in contact with an alkaline solution

Journal of nuclear materials, 2002-12, Vol.306 (2), p.99-104

2002

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Study of zircaloy-4 fuel cladding corrosion using ion beams.: Application to long-term disposal of nuclear wastes

Nuclear instruments & methods in physics research. Section B, Beam interactions with materials and atoms, 2001-07, Vol.181 (1), p.640-643

2001

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G.P.43: Gene replacement therapy of myotubular myopathy: Restricting expression of MTM1 in skeletal muscle

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9-10), p.807-808

2014

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T.O.13 - Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S209-S209

2016

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G.P.41: Canine X-linked myotubular myopathy: A dose-finding study of systemic AAV8-MTM1 effects on muscle strength, gait, respiration, neuromuscular function and survival

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9-10), p.807-807

2014

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G.P.294 - Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S274-S274

2015

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Use of RBS to validate europium surface implantation as a marker for the corrosion study of zirconia

Nuclear Instruments and Methods in Physics Research Section B: Beam Interactions with Materials and Atoms, 2000, Vol.161, p.668-672

2000

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Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid

Virology (New York, N.Y.), 2003-05, Vol.309 (2), p.203-208

2003

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Optimised helper virus-free production of high-quality adeno-associated virus vectors

The journal of gene medicine, 2001-01, Vol.3 (1), p.59-71

2001

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Study of zircaloy-4 fuel cladding corrosion using ion beams

Nuclear instruments & methods in physics research. Section B, Beam interactions with materials and atoms, 2001-07, Vol.181 (1-4), p.640-643

2001

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Intracellular Trafficking of Adeno-Associated Virus Vectors: Routing to the Late Endosomal Compartment and Proteasome Degradation

Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

G.P.43

G.P.41

Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

P.4.3 Intravenous infusion of AAV8–MTM1 prolongs life and ameliorates severe muscle pathology in mouse and dog models of X-linked myotubular myopathy

AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy

P1.44 Neuromuscular junction abnormalities and myotubular myopathy

Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice

T.O.4 Development of AAV-gene and protein-based therapies for X-linked myotubular myopathy

CONGENITAL MYOPATHIES – NEMALINE MYOPATHIES: EP.40 Neuromuscular junction defects in ACTA1-related nemaline myopathy

CONGENITAL MYOPATHIES – NEMALINE MYOPATHIES

O.32Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in muscle of DM1 mice

CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1

P.278 - CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1

Iodine release from oxidised Zircaloy cladding in contact with an alkaline solution

Study of zircaloy-4 fuel cladding corrosion using ion beams.: Application to long-term disposal of nuclear wastes

G.P.43: Gene replacement therapy of myotubular myopathy: Restricting expression of MTM1 in skeletal muscle

T.O.13 - Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

G.P.41: Canine X-linked myotubular myopathy: A dose-finding study of systemic AAV8-MTM1 effects on muscle strength, gait, respiration, neuromuscular function and survival

G.P.294 - Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

Use of RBS to validate europium surface implantation as a marker for the corrosion study of zirconia

Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid

Optimised helper virus-free production of high-quality adeno-associated virus vectors

Study of zircaloy-4 fuel cladding corrosion using ion beams

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