In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization
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
Published in Nucleic acids research (15.10.2015)
Published in Nucleic acids research (15.10.2015)
Get full text
Journal Article
Induced secretion of β-hexosaminidase by human brain endothelial cells: A novel approach in Sandhoff disease?
Batista, Lionel, Miller, Florence, Clave, Céline, Arfi, Audrey, Douillard-Guilloux, Gaëlle, Couraud, Pierre-Olivier, Caillaud, Catherine
Published in Neurobiology of disease (01.03.2010)
Published in Neurobiology of disease (01.03.2010)
Get full text
Journal Article
New insights into therapeutic options for Pompe disease
Richard, Emmanuel, Douillard-Guilloux, Gaëlle, Caillaud, Catherine
Published in IUBMB life (01.11.2011)
Published in IUBMB life (01.11.2011)
Get more information
Journal Article
Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease
Douillard-Guilloux, Gaëlle, Richard, Emmanuel, Batista, Lionel, Caillaud, Catherine
Published in The journal of gene medicine (01.04.2009)
Published in The journal of gene medicine (01.04.2009)
Get full text
Journal Article
Immortalization of murine muscle cells from lysosomal α-glucosidase deficient mice: A new tool to study pathophysiology and assess therapeutic strategies for Pompe disease
Douillard-Guilloux, Gaëlle, Mouly, Vincent, Caillaud, Catherine, Richard, Emmanuel
Published in Biochemical and biophysical research communications (16.10.2009)
Published in Biochemical and biophysical research communications (16.10.2009)
Get full text
Journal Article
Molecular evidence for BDNF- and GABA-related dysfunctions in the amygdala of female subjects with major depression
GUILLOUX, J.-P, DOUILLARD-GUILLOUX, G, KOTA, R, WANG, X, GARDIER, A. M, MARTINOWICH, K, TSENG, G. C, LEWIS, D. A, SIBILLE, E
Published in Molecular psychiatry (01.11.2012)
Published in Molecular psychiatry (01.11.2012)
Get full text
Journal Article
Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II
Douillard-Guilloux, Gaelle, Raben, Nina, Takikita, Shoichi, Batista, Lionel, Caillaud, Catherine, Richard, Emmanuel
Published in Human molecular genetics (15.12.2008)
Published in Human molecular genetics (15.12.2008)
Get full text
Journal Article
Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease
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
Published in Human molecular genetics (15.02.2010)
Published in Human molecular genetics (15.02.2010)
Get full text
Journal Article
Anticipated Brain Molecular Aging in Major Depression
Douillard-Guilloux, Gaëlle, Guilloux, Jean-Philippe, Lewis, David A., Sibille, Etienne
Published in The American journal of geriatric psychiatry (01.10.2012)
Published in The American journal of geriatric psychiatry (01.10.2012)
Get full text
Journal Article
Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of [alpha]-glucosidase in Pompe disease
Douillard-Guilloux, Gaëlle, Richard, Emmanuel, Batista, Lionel, Caillaud, Catherine
Published in The journal of gene medicine (01.04.2009)
Published in The journal of gene medicine (01.04.2009)
Get full text
Journal Article