In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization von Kamola, Piotr J, Kitson, Jeremy D A, Turner, Gemma, Maratou, Klio, Eriksson, Sofie, Panjwani, Aliza, Warnock, Linda C, Douillard Guilloux, Gaelle A, Moores, Kitty, Koppe, Emma L, Wixted, William E, Wilson, Paul A, Gooderham, Nigel J, Gant, Timothy W, Clark, Kenneth L, Hughes, Stephen A, Edbrooke, Mark R, Parry, Joel D

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Anticipated Brain Molecular Aging in Major Depression von Douillard-Guilloux, Gaëlle, Ph.D, Guilloux, Jean-Philippe, Ph.D, Lewis, David A., M.D, Sibille, Etienne, Ph.D

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Brain molecular aging, promotion of neurological disease and modulation by Sirtuin5 longevity gene polymorphism von Glorioso, Christin, Oh, Sunghee, Douillard, Gaelle Guilloux, Sibille, Etienne

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Decrease in somatostatin‐positive cell density in the amygdala of females with major depression von Douillard‐Guilloux, Gaelle, Lewis, David, Seney, Marianne L., Sibille, Etienne

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Induced secretion of β-hexosaminidase by human brain endothelial cells: A novel approach in Sandhoff disease? von Batista, Lionel, Miller, Florence, Clave, Céline, Arfi, Audrey, Douillard-Guilloux, Gaëlle, Couraud, Pierre-Olivier, Caillaud, Catherine

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New insights into therapeutic options for Pompe disease von Richard, Emmanuel, Douillard‐Guilloux, Gaëlle, Caillaud, Catherine

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Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease von Douillard-Guilloux, Gaëlle, Richard, Emmanuel, Batista, Lionel, Caillaud, Catherine

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Disruption of the proprioceptive mapping in the medial wall of parkinsonian monkeys von Escola, Ludovic, Michelet, Thomas, Douillard, Gaelle, Guehl, Dominique, Bioulac, Bernard, Burbaud, Pierre

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Immortalization of murine muscle cells from lysosomal α-glucosidase deficient mice: A new tool to study pathophysiology and assess therapeutic strategies for Pompe disease von Douillard-Guilloux, Gaëlle, Mouly, Vincent, Caillaud, Catherine, Richard, Emmanuel

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Molecular evidence for BDNF- and GABA-related dysfunctions in the amygdala of female subjects with major depression von Guilloux, J-P, Douillard-Guilloux, G, Kota, R, Wang, X, Gardier, A M, Martinowich, K, Tseng, G C, Lewis, D A, Sibille, E

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Correction of glycogenosis type 2 by muscle-specific lentiviral vector von Richard, Emmanuel, Douillard-Guilloux, Gaëlle, Batista, Lionel, Caillaud, Catherine

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Decrease in somatostatin-positive cell density in the amygdala of females with major depression: Douillard-Guilloux et al von Douillard-Guilloux, Gaelle, Lewis, David, Seney, Marianne L., Sibille, Etienne

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Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II von Douillard-Guilloux, Gaelle, Raben, Nina, Takikita, Shoichi, Batista, Lionel, Caillaud, Catherine, Richard, Emmanuel

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910. Gene Therapy of Glycogenosis Type 2Using SIN-Lentiviral Vectors von Emmanuel, Richard, Safa, Benhassine, Gaelle, Douillard, Lionel, Batista, Catherine, Caillaud

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Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease von Douillard-Guilloux, Gaelle, Raben, Nina, Takikita, Shoichi, Ferry, Arnaud, Vignaud, Alban, Guillet-Deniau, Isabelle, Favier, Maryline, Thurberg, Beth L., Roach, Peter J., Caillaud, Catherine, Richard, Emmanuel

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Brain molecular aging, promotion of neurological disease and modulation by sirtuin 5 longevity gene polymorphism von Glorioso, Christin, Oh, Sunghee, Douillard, Gaelle Guilloux, Sibille, Etienne

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Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation von Richard, Emmanuel, Douillard-Guilloux, Gaelle, Caillaud, Catherine

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New insights into therapeutic options for Pompe disease von Richard, Emmanuel, Douillard‐Guilloux, Gaëlle, Caillaud, Catherine

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Anticipated Brain Molecular Aging in Major Depression von Douillard-Guilloux, Gaëlle, Guilloux, Jean-Philippe, Lewis, David A., Sibille, Etienne

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Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of [alpha]-glucosidase in Pompe disease von Douillard-Guilloux, Gaëlle, Richard, Emmanuel, Batista, Lionel, Caillaud, Catherine

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