Propagation of Distinct α-Synuclein Strains Within Human Reconstructed Neuronal Network and Associated Neuronal Dysfunctions
Simona Gribaudo
,
Luc Bousset
,
Josquin Courte
,
Ronald Melki
,
Jean-Michel Peyrin
et al.
Book sections
hal-03836488v1
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Toxic CUG RNA repeats disrupt developmentally-regulated splicing in oligodendrocytes causing transient hypomyelination in a mouse model of myotonic dystrophy.
Louison Lallemant
,
Sandra Braz
,
Anchel González-Barriga
,
Paul Magneron
,
Aurélien Cordier
et al.
The European Meeting on Glial Cells in Health and Disease , Jul 2023, Berlin, France
Conference poster
hal-04005524v1
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Ex vivo gene therapy for β-hemoglobinpathies and metabolic disorders
Marine Laurent
Theses
tel-04021549v1
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MBNL‐dependent impaired development within the neuromuscular system in myotonic dystrophy type 1
Julie Tahraoui-Bories
,
Antoine Mérien
,
Anchel González-Barriga
,
Jeanne Lainé
,
Céline Leteur
et al.
Journal articles
hal-03992575v1
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Photoreceptor Cell Replacement Using Pluripotent Stem Cells: Current Knowledge and Remaining Questions
Christelle Monville
,
Olivier Goureau
,
Karim Ben M'Barek
Cold Spring Harbor Perspectives in Medicine , 2022
Journal articles
hal-03932669v1
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Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
Clément Pontoizeau
,
Marcelo Simon-Sola
,
Clovis Gaborit
,
Vincent Nguyen
,
Irina Rotaru
et al.
Journal articles
hal-03862521v1
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Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes
Diana Dincã
,
Anchel González-Barriga
,
Geraldine Sicot
,
Louison Lallemant
,
Laure-Elise Pillet
et al.
2022
Preprints, Working Papers, ...
hal-03863376v1
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Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
Panagiotis Antoniou
,
Giulia Hardouin
,
Pierre Martinucci
,
Giacomo Frati
,
Tristan Felix
et al.
Journal articles
mnhn-03868573v1
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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
et al.
Journal articles
hal-03454735v1
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Severe congenital myasthenic syndromes caused by agrin mutations affecting secretion by motoneurons
Arnaud Jacquier
,
Valérie Risson
,
Thomas Simonet
,
Florine Roussange
,
Nicolas Lacoste
et al.
Journal articles
hal-03863096v1
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Immune Responses to Gene Editing by Viral and Non-Viral Delivery Vectors Used in Retinal Gene Therapy
Duohao Ren
,
Sylvain Fisson
,
Deniz Dalkara
,
Divya Ail
Journal articles
inserm-03806869v1
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MBNL dependent-impaired development connectivity within neuromuscular circuits in Myotonic Dystrophy type.
Julie Tahraoui-Bories
,
Antoine Mérien
,
Jeanne Lainé
,
Florine Roussange
,
Anchel González-Barriga
et al.
Myology 2022 , Sep 2022, Nice, France
Conference poster
hal-04005593v1
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Glial cell dysfunction in myotonic dystrophy brain disease
Dm Dinca
,
So Braz
,
Louison Lallemant
,
A Gonzalez-Barriga
,
B Potier
et al.
12th Japanese-French Workshop: New insights in personalized medicine for neuromuscular diseases: From Basic to Applied Myology , Sep 2022, Giverny, France
Conference papers
hal-04004421v1
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RNA helicase-dependent gene looping impacts messenger RNA processing
Sophie Terrone
,
Jessica Valat
,
Nicolas Fontrodona
,
Guillaume Giraud
,
Jean-Baptiste Claude
et al.
Journal articles
hal-03842696v1
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Modélisation des atteintes neurologiques du syndrome de Wolfram de type 1 : exploitation des cellules souches pluripotentes induites et de leurs dérivés
Axel Sciauvaud
Theses
tel-03917165v1
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Human Cytomegalovirus Modifies Placental Small Extracellular Vesicle Composition to Enhance Infection of Fetal Neural Cells In Vitro
Mathilde Bergamelli
,
Hélène Martin
,
Yann Aubert
,
Jean-Michel Mansuy
,
Marlène Marcellin
et al.
Journal articles
hal-03800516v1
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Moxifloxacin rescues SMA phenotypes in patient-derived cells and animal model
Camille Januel
,
Giovanna Menduti
,
Kamel Mamchaoui
,
Cecile Martinat
,
Ruben Artero
et al.
Journal articles
hal-03832679v1
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Deciphering the Molecular Mechanism of Incurable Muscle Disease by a Novel Method for the Interpretation of miRNA Dysregulation
David Israeli
,
Ai Vu Hong
,
Guillaume Corre
,
Quentin Miagoux
,
Isabelle Richard
et al.
Journal articles
hal-03874746v1
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MELAS syndrome causes glutamate and tricarboxylic acid cycle dysfunctions alleviated by ketone body treatment
Sophie Belal
,
David Goudenège
,
Cinzia Bocca
,
Florent Dumont
,
Juan Manuel Chao de la Barca
et al.
Journal articles
hal-03861087v1
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MBNL-dependent impaired development connectivity within neuromuscular circuits in myotonic dystrophy type 1
Julie Tahraoui-Bories
,
Antoine Mérien
,
Florine Roussange
,
Anchel González-Barriga
,
Jeanne Lainé
et al.
International Myotonic Dystrophy Consortium Meeting , Jun 2022, Osaka, Japan
Conference poster
hal-04006954v1
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Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes
Diana Mihaela Dincã
,
Louison Lallemant
,
Anchel González-Barriga
,
Noemie Cresto
,
Sandra Braz
et al.
International Myotonic Dystrophy Consortium Meeting , Jun 2022, Osaka, Japan
Conference poster
hal-04006873v1
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Target-agnostic drug discovery approach using informative high-content imaging for identification of a myogenic modulator in DM1 context
Florine Roussange
,
Amélie Weiss
,
Kalina Radoynovska
,
Jacqueline Gide
,
Johana Tournois
et al.
International Myotonic Dystrophy Consortium Meeting IDMC-13 , May 2022, Osaka, Japan
Conference poster
hal-04022935v1
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MN dependent-impaired development connectivity within neuromuscular circuits in Myotonic Dystrophy type 1
Julie Tahraoui-Bories
,
Antoine Mérien
,
Florine Roussange
,
Anchel Gonzalez-Barriga
,
Jeanne Lainé
et al.
International Myotonic Dystrophy Consortium Meeting IDMC-13 , May 2022, Osaka, Japan
Conference poster
hal-04022921v1
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Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination
Lucile Hoch
,
Nathalie Bourg
,
Fanny Degrugillier
,
Céline Bruge
,
Manon Benabides
et al.
Journal articles
hal-03874719v1
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Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients
David-Alexandre Gross
,
Novella Tedesco
,
Christian Leborgne
,
Giuseppe Ronzitti
Journal articles
hal-03640310v1
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Systemic and local immune responses to intraocular AAV vector administration in non-human primates
Divya Ail
,
Duohao Ren
,
Elena Brazhnikova
,
Céline Nouvel-Jaillard
,
Stephane Bertin
et al.
Journal articles
hal-03602826v1
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Cytokines, chemokines and growth factors profile in human aqueous humor in idiopathic uveitis
Marie-Hélène Errera
,
Ana Pratas
,
Sylvain Fisson
,
Thomas Manicom
,
Marouane Boubaya
et al.
Journal articles
hal-03568725v1
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Generation of a heterozygous SCN5A knockout human induced pluripotent stem cell line by CRISPR/Cas9 edition
Marie Gizon
,
Laëtitia Duboscq-Bidot
,
Lina El Kassar
,
Pierre Bobin
,
Flavie Ader
et al.
Journal articles
hal-03549731v1
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CRISPR gene editing in pluripotent stem cells reveals the function of MBNL proteins during human in vitro myogenesis
Antoine Mérien
,
Julie Tahraoui-Bories
,
Michel Cailleret
,
Jean-Baptiste Dupont
,
Céline Leteur
et al.
Journal articles
hal-03830948v1
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Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes
Diana Dincã
,
Louison Lallemant
,
Anchel González-Barriga
,
Noémie Cresto
,
Sandra Braz
et al.
Journal articles
hal-03715682v1
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