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Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy
Mariot V, Le Guiner C, Barthelemy I, et al.
Molecular therapy. Methods & clinical development, 2020, 18, p 415
An AAV-SGCG Dose-Response Study in a gamma-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.
Israeli D, Cosette J, Corre G, et al.
Molecular therapy. Methods & clinical development, 2019, 13, p 494
RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
Domenger C, Allais M, François V, et al.
Molecular therapy. Nucleic acids, 2018, 10, p 277
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Le Guiner C, Servais L, Montus M, et al.
Nature communications, 2017, 8
Non-Ambulant Duchenne Patients Theoretically treatable by Exon 53 Skipping have severe Phenotype
Servais L, Montus M, Le Guiner C, et al.
Journal of Neuromuscular Diseases, 2015, 2, 3, p 269
Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy
Wary C, Azzabou N, Giraudeau C, et al.
NMR in biomedicine, 2015, 28, 9, p 1150
Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients
Le Guiner C, Montus M, Servais L, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2014, 22, 11, p 1923
Distinctive serum miRNA profile in mouse models of striated muscular pathologies
Vignier N, Amor F, Fogel P, et al.
PLoS ONE, 2013, 8, 2, e55281, 14 p.
A phase I trial of adeno-associated virus serotype 1-gamma-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
Herson S, Hentati F, Rigolet A, et al.
Brain, 2012, 135, 2, p 483
Biodistribution and shedding of RAAV8-U7 SNRNA vectors after locoregional injection in the forelimb of GRMD dogs
François V, Le Guiner C, Montus M, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 88
Development of a dynamometer for the forelimb strength assessment in GRMD dogs (golden retriever muscular dystrophy)
Hogrel JY, Moraux A, Costiou P, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 133
Dystrophin expression after locoregional injection of RAAV8-U7 SNRNA vectors in the forelimb of GRMD dogs
Cherel Y, Larcher T, François V, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 78
Forelimb loco-regional injection of RAAV8-U7SNRNA in GRMD dogs allows dose-dependant dystrophin expression and phenotypic correction
Servais L, Le Guiner C, Montus M, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 25
Intra-muscular distribution of dystrophin positive fibers after locoregional injection of RAAV8-U7 SNRNA vectors in thefore limb of GRMD dogs
Cherel Y, Larcher T, Deschamps JY, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 38
A phase I dose-escalating trial of AAV1--sarcoglycan gene therapy for limb girdle muscular dystrophy type 2c
Benveniste O, Hentati F, Rigolet A, et al.
Congrès : 4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France))
2011, p. 25
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models
Marangoni F, Bosticardo M, Charrier S, et al.
Current opinion in molecular therapeutics, 2009, 17, 6, p. 1073-1082
Correction of muscle function in myotubular myopathy by AAV-mediated MTM1 replacement
Buj Bello A, Fougerousse F, Jamet T, et al.
Congrès : Congrès international de myologie 2008 (International Congress of Myology 2008; 26-30 mai 2008; Marseille, France)
2008, p. 445
AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis
Buj-Bello A, Fougerousse F, Schwab Y, et al.
Human molecular genetics, 2008, 17, 14, p. 2132-2143
