Search Results - "KLEIN, Arnaud"

Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice by Lo Scrudato, Mirella, Poulard, Karine, Sourd, Célia, Tomé, Stéphanie, Klein, Arnaud F., Corre, Guillaume, Huguet, Aline, Furling, Denis, Gourdon, Geneviève, Buj-Bello, Ana

“…Myotonic dystrophy type 1 (DM1) is caused by a CTG repeat expansion located in the 3′ UTR of the DMPK gene. Expanded DMPK transcripts aggregate into nuclear…”
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Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes by Dastidar, Sumitava, Majumdar, Debanjana, Tipanee, Jaitip, Singh, Kshitiz, Klein, Arnaud F., Furling, Denis, Chuah, Marinee K., VandenDriessche, Thierry

“…CTG repeat expansion (CTGexp) is associated with aberrant alternate splicing that contributes to cardiac dysfunction in myotonic dystrophy type 1 (DM1)…”
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The beneficial effect of chronic muscular exercise on muscle fragility is increased by Prox1 gene transfer in dystrophic mdx muscle by Monceau, Alexandra, Delacroix, Clément, Lemaitre, Mégane, Revet, Gaelle, Furling, Denis, Agbulut, Onnik, Klein, Arnaud, Ferry, Arnaud

“…Greater muscle fragility is thought to cause the exhaustion of the muscle stem cells during successive degeneration/repair cycles, leading to muscle wasting…”
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Dystrophin Restoration after Adeno-Associated Virus U7–Mediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model by Monceau, Alexandra, Moutachi, Dylan, Lemaitre, Mégane, Garcia, Luis, Trollet, Capucine, Furling, Denis, Klein, Arnaud, Ferry, Arnaud

“…Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease caused by Dmd mutations, resulting in the absence of dystrophin in skeletal muscle, and a…”
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Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice by Klein, Arnaud F, Varela, Miguel A, Arandel, Ludovic, Holland, Ashling, Naouar, Naira, Arzumanov, Andrey, Seoane, David, Revillod, Lucile, Bassez, Guillaume, Ferry, Arnaud, Jauvin, Dominic, Gourdon, Genevieve, Puymirat, Jack, Gait, Michael J, Furling, Denis, Wood, Matthew Ja

“…Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including myotonic dystrophy…”
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Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells by Dastidar, Sumitava, Ardui, Simon, Singh, Kshitiz, Majumdar, Debanjana, Nair, Nisha, Fu, Yanfang, Reyon, Deepak, Samara, Ermira, Gerli, Mattia F M, Klein, Arnaud F, De Schrijver, Wito, Tipanee, Jaitip, Seneca, Sara, Tulalamba, Warut, Wang, Hui, Chai, Yoke Chin, In't Veld, Peter, Furling, Denis, Tedesco, Francesco Saverio, Vermeesch, Joris R, Joung, J Keith, Chuah, Marinee K, VandenDriessche, Thierry

“…Abstract CRISPR/Cas9 is an attractive platform to potentially correct dominant genetic diseases by gene editing with unprecedented precision. In the current…”
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Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds by Arandel, Ludovic, Polay Espinoza, Micaela, Matloka, Magdalena, Bazinet, Audrey, De Dea Diniz, Damily, Naouar, Naïra, Rau, Frédérique, Jollet, Arnaud, Edom-Vovard, Frédérique, Mamchaoui, Kamel, Tarnopolsky, Mark, Puymirat, Jack, Battail, Christophe, Boland, Anne, Deleuze, Jean-Francois, Mouly, Vincent, Klein, Arnaud F, Furling, Denis

“…Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are autosomal dominant neuromuscular diseases caused by microsatellite expansions and belong to the family of…”
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Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy by Fugier, Charlotte, Klein, Arnaud F, Hammer, Caroline, Vassilopoulos, Stéphane, Ivarsson, Ylva, Toussaint, Anne, Tosch, Valérie, Vignaud, Alban, Ferry, Arnaud, Messaddeq, Nadia, Kokunai, Yosuke, Tsuburaya, Rie, de la Grange, Pierre, Dembele, Doulaye, Francois, Virginie, Precigout, Guillaume, Boulade-Ladame, Charlotte, Hummel, Marie-Christine, de Munain, Adolfo Lopez, Sergeant, Nicolas, Laquerrière, Annie, Thibault, Christelle, Deryckere, François, Auboeuf, Didier, Garcia, Luis, Zimmermann, Pascale, Udd, Bjarne, Schoser, Benedikt, Takahashi, Masanori P, Nishino, Ichizo, Bassez, Guillaume, Laporte, Jocelyn, Furling, Denis, Charlet-Berguerand, Nicolas

“…Myotonic dystrophy is a slowly progressing muscle disease marked by muscle wasting and myotonia. Nicolas Charlet-Berguerand and his colleagues have found that…”
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Improvement of Dystrophic Muscle Fragility by Short-Term Voluntary Exercise through Activation of Calcineurin Pathway in mdx Mice by Delacroix, Clement, Hyzewicz, Janek, Lemaitre, Megane, Friguet, Bertrand, Li, Zhenlin, Klein, Arnaud, Furling, Denis, Agbulut, Onnik, Ferry, Arnaud

“…Dystrophin deficiency in mdx mice, a model for Duchenne muscular dystrophy, leads to muscle weakness revealed by a reduced specific maximal force as well as…”
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Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs by Furling, Denis, François, Virginie, Klein, Arnaud F, Beley, Cyriaque, Jollet, Arnaud, Lemercier, Camille, Garcia, Luis

“…We describe a function for modified human U7 small nuclear RNAs (hU7-snRNAs) distinct from modification of pre-mRNA splicing events. Engineered hU7-snRNAs…”
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Cells of Matter- In Vitro Models for Myotonic Dystrophy by Matloka, Magdalena, Klein, Arnaud F, Rau, Frédérique, Furling, Denis

“…Myotonic dystrophy type 1 (DM1 also known as Steinert disease) is a multisystemic disorder mainly characterized by myotonia, progressive muscle weakness and…”
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Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells by Porquet, Florent, Weidong, Lin, Jehasse, Kévin, Gazon, Hélène, Kondili, Maria, Blacher, Silvia, Massotte, Laurent, Di Valentin, Emmannuel, Furling, Denis, Gillet, Nicolas Albert, Klein, Arnaud François, Seutin, Vincent, Willems, Luc

“…Myotonic dystrophy type 1 (DM1) is a neuromuscular disease that originates from an expansion of CTG microsatellites in the 3′ untranslated region of the DMPK…”
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New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats by Laurent, François-Xavier, Sureau, Alain, Klein, Arnaud F, Trouslard, François, Gasnier, Erwan, Furling, Denis, Marie, Joëlle

“…Myotonic Dystrophy type I (DM1) is caused by an abnormal expansion of CTG triplets in the 3' UTR of the dystrophia myotonica protein kinase (DMPK) gene,…”
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Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats by Arandel, Ludovic, Matloka, Magdalena, Klein, Arnaud F., Rau, Frédérique, Sureau, Alain, Ney, Michel, Cordier, Aurélien, Kondili, Maria, Polay-Espinoza, Micaela, Naouar, Naira, Ferry, Arnaud, Lemaitre, Mégane, Begard, Séverine, Colin, Morvane, Lamarre, Chloé, Tran, Hélène, Buée, Luc, Marie, Joëlle, Sergeant, Nicolas, Furling, Denis

“…Myotonic dystrophy type 1 (DM1) is an RNA-dominant disease whose pathogenesis stems from the functional loss of muscleblind-like RNA-binding proteins (RBPs),…”
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Large CTG Repeats Trigger p16-Dependent Premature Senescence in Myotonic Dystrophy Type 1 Muscle Precursor Cells by Bigot, Anne, Klein, Arnaud F, Gasnier, Erwan, Jacquemin, Virginie, Ravassard, Philippe, Butler-Browne, Gillian, Mouly, Vincent, Furling, Denis

“…A CTG repeat amplification is responsible for the dominantly inherited neuromuscular disorder, myotonic dystrophy type 1 (DM1), which is characterized by…”
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PABPN1 overexpression leads to upregulation of genes encoding nuclear proteins that are sequestered in oculopharyngeal muscular dystrophy nuclear inclusions by Corbeil-Girard, Louis-Philippe, Klein, Arnaud F., Sasseville, A. Marie-Josée, Lavoie, Hugo, Dicaire, Marie-Josée, Saint-Denis, Anik, Pagé, Martin, Duranceau, André, Codère, François, Bouchard, Jean-Pierre, Karpati, George, Rouleau, Guy A., Massie, Bernard, Langelier, Yves, Brais, Bernard

“…Oculopharyngeal muscular dystrophy (OPMD) is an adult-onset disease caused by expanded (GCN) 12–17 stretches encoding the N-terminal polyalanine domain of the…”
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PABPN1 polyalanine tract deletion and long expansions modify its aggregation pattern and expression by Klein, Arnaud F., Ebihara, Mitsuru, Alexander, Christine, Dicaire, Marie-Josée, Sasseville, A. Marie-Josée, Langelier, Yves, Rouleau, Guy A., Brais, Bernard

“…Expansions of a (GCN) 10/polyalanine tract in the Poly(A) Binding Protein Nuclear 1 (PABPN1) cause autosomal dominant oculopharyngeal muscular dystrophy…”
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The dynamism of PABPN1 nuclear inclusions during the cell cycle by Marie-Josée Sasseville, A., Caron, Antoine W., Bourget, Lucie, Klein, Arnaud F., Dicaire, Marie-Josée, Rouleau, Guy A., Massie, Bernard, Langelier, Yves, Brais, Bernard

“…Oculopharyngeal muscular dystrophy (OPMD) is caused by expansion of a (GCN)10 to a (GCN)11–17 repeat coding for a polyalanine domain at the N-terminal part of…”
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PGN-EDODM1: Preclinical Data Supporting the Development of an Enhanced Delivery Oligonucleotide (EDO) for the Treatment of Myotonic Dystrophy Type 1 (DM1) (S48.001) by Holland, Ashling, Klein, Arnaud, Godfrey, Caroline, Svenstrup, Niels, Larkindale, Jane, Bracegirdle, Sonia, Furling, Denis, Goyal, Jaya

“…Abstract only…”
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Dystrophin Restoration after Adeno-Associated Virus U7–Mediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model by Monceau, Alexandra, Moutachi, Dylan, Lemaitre, Mégane, Garcia, Luis, Trollet, Capucine, Furling, Denis, Klein, Arnaud, Ferry, Arnaud

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