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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice
P. Sellier
,
P. Vidal
,
B. Bertin
,
E. Gicquel
,
E Bertil‐froidevaux
Article dans une revue
hal-04302850v1
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Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular Dystrophies
Celine Bruge
,
Marine Geoffroy
,
Manon Benabides
,
Emilie Pellier
,
Evelyne Gicquel
Article dans une revue
hal-03858917v1
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Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in Duchenne muscular dystrophy
Ai Vu Hong
,
Nathalie Bourg
,
Peggy Sanatine
,
Jerome Poupiot
,
Karine Charton
Article dans une revue
hal-03454735v2
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Minimal Consequences of CMAH and DBA/2J Background on a FKRP Deficient Model
Camille Vaubourg
,
Evelyne Gicquel
,
Isabelle Richard
,
William Lostal
,
Jessica Bellec
Article dans une revue
hal-03003866v2
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Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants
Sara F Henriques
,
Evelyne Gicquel
,
Justine Marsolier
,
Isabelle Richard
Article dans une revue
hal-02177499v1
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AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression
Evelyne Gicquel
,
Natacha Maizonnier
,
Steven Foltz
,
William Martin
,
Nathalie Bourg
Article dans une revue
hal-02177575v1
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γ-sarcoglycan and dystrophin mutation spectrum in an Algerian cohort
Evelyne Gicquel
,
Natacha Maizonnier
,
Steven Foltz
,
William Martin
,
Nathalie Bourg
Article dans une revue
hal-02333075v1
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216th ENMC international workshop: Clinical readiness in FKRP related myopathies January 15–17, 2016 Naarden, The Netherlands
Isabelle Richard
,
Jean-Pierre Laurent
,
Sebahattin Cirak
,
John Vissing
,
Susan Brown
Article dans une revue
hal-02333077v1
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The Phenotype of Dysferlin-Deficient Mice Is Not Rescued by Adeno-Associated Virus–Mediated Transfer of Anoctamin 5
Florence Le Roy
,
Laurence Suel
,
Jérôme Poupiot
,
Marc Bartoli
,
François Monjaret
Article dans une revue
hal-02336935v1
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A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome.
Gaëlle Blandin
,
Sylvie Marchand
,
Karine Charton
,
Nathalie Danièle
,
Evelyne Gicquel
Article dans une revue
inserm-00805816v1
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Rescue of Sarcoglycan Mutations by Inhibition of Endoplasmic Reticulum Quality Control is Associated with Minimal Structural Modifications
Tayebeh Soheili
,
Evelyne Gicquel
,
Jérôme Poupiot
,
Luu N'Guyen
,
Florence Le Roy
Article dans une revue
hal-01610024v1
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Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies
Karine Charton
,
Nathalie Danièle
,
Anna Vihola
,
Carinne Roudaut
,
Evelyne Gicquel
Article dans une revue
hal-02321460v1
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Mannosidase I inhibition rescues the human alpha-sarcoglycan R77C recurrent mutation.
Marc Bartoli
,
Evelyne Gicquel
,
Laetitia Barrault
,
Tayebeh Soheili
,
Marie Malissen
Article dans une revue
hal-00294192v1
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NF-kappa B-dependent expression of the antiapoptotic factor c-FLIP is regulated by calpain 3, the protein involved in limb-girdle muscular dystrophy type 2A
Beatrice Benayoun
,
Stephen Baghdiguian
,
Alicia Lajmanovich
,
Marc Bartoli
,
Nathalie Danièle
Article dans une revue
hal-01610041v1
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Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector
Françoise Fougerousse
,
Marc Bartoli
,
Jérôme Poupiot
,
Ludovic Arandel
,
Muriel Durand
Article dans une revue
hal-01610043v1
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Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2A
Marc Bartoli
,
C Roudaut
,
S Martin
,
F Fougerousse
,
L Suel
Article dans une revue
hal-01610047v1
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