Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Le Guiner, Caroline, Servais, Laurent, Montus, Marie, Larcher, Thibaut, Fraysse, Bodvaël, Moullec, Sophie, Allais, Marine, François, Virginie, Dutilleul, Maeva, Malerba, Alberto, Koo, Taeyoung, Thibaut, Jean-Laurent, Matot, Béatrice, Devaux, Marie, Le Duff, Johanne, Deschamps, Jack-Yves, Barthelemy, Inès, Blot, Stéphane, Testault, Isabelle, Wahbi, Karim, Ederhy, Stéphane, Martin, Samia, Veron, Philippe, Georger, Christophe, Athanasopoulos, Takis, Masurier, Carole, Mingozzi, Federico, Carlier, Pierre, Gjata, Bernard, Hogrel, Jean-Yves, Adjali, Oumeya, Mavilio, Fulvio, Voit, Thomas, Moullier, Philippe, Dickson, George
Published in Nature communications (25.07.2017)
Published in Nature communications (25.07.2017)
Get full text
Journal Article
Web Resource
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Cirak, Sebahattin, MD, Arechavala-Gomeza, Virginia, PhD, Guglieri, Michela, MD, Feng, Lucy, PhD, Torelli, Silvia, PhD, Anthony, Karen, PhD, Abbs, Stephen, PhD, Garralda, Maria Elena, Prof, Bourke, John, MD, Wells, Dominic J, Prof, Dickson, George, Prof, Wood, Matthew JA, MD PhD, Wilton, Steve D, Prof, Straub, Volker, Prof, Kole, Ryszard, Prof, Shrewsbury, Stephen B, MD, Sewry, Caroline, Prof, Morgan, Jennifer E, PhD, Bushby, Kate, Prof, Muntoni, Francesco, Prof
Published in The Lancet (British edition) (13.08.2011)
Published in The Lancet (British edition) (13.08.2011)
Get full text
Journal Article
Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9
Koo, Taeyoung, Lu-Nguyen, Ngoc B., Malerba, Alberto, Kim, Eunji, Kim, Daesik, Cappellari, Ornella, Cho, Hee-Yeon, Dickson, George, Popplewell, Linda, Kim, Jin-Soo
Published in Molecular therapy (06.06.2018)
Published in Molecular therapy (06.06.2018)
Get full text
Journal Article
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Kinali, Maria, MD, Arechavala-Gomeza, Virginia, PhD, Feng, Lucy, PhD, Cirak, Sebahattin, MD, Hunt, David, FRCS, Adkin, Carl, PhD, Guglieri, Michela, MD, Ashton, Emma, PhD, Abbs, Stephen, PhD, Nihoyannopoulos, Petros, MD, Garralda, Maria Elena, MD, Rutherford, Mary, MD, Mcculley, Caroline, PhD, Popplewell, Linda, PhD, Graham, Ian R, PhD, Dickson, George, PhD, Wood, Matthew JA, MD, Wells, Dominic J, PhD, Wilton, Steve D, PhD, Kole, Ryszard, PhD, Straub, Volker, MD, Bushby, Kate, MD, Sewry, Caroline, PhD, Morgan, Jennifer E, PhD, Muntoni, Francesco, MD
Published in Lancet neurology (01.10.2009)
Published in Lancet neurology (01.10.2009)
Get full text
Journal Article
Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
Zhou, Haiyan, Meng, Jinhong, Malerba, Alberto, Catapano, Francesco, Sintusek, Palittiya, Jarmin, Susan, Feng, Lucy, Lu‐Nguyen, Ngoc, Sun, Lianwen, Mariot, Virginie, Dumonceaux, Julie, Morgan, Jennifer E., Gissen, Paul, Dickson, George, Muntoni, Francesco
Published in Journal of cachexia, sarcopenia and muscle (01.06.2020)
Published in Journal of cachexia, sarcopenia and muscle (01.06.2020)
Get full text
Journal Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Pichavant, Christophe, Aartsma-Rus, Annemieke, Clemens, Paula R, Davies, Kay E, Dickson, George, Takeda, Shin'ichi, Wilton, Steve D, Wolff, Jon A, Wooddell, Christine I, Xiao, Xiao, Tremblay, Jacques P
Published in Molecular therapy (01.05.2011)
Published in Molecular therapy (01.05.2011)
Get full text
Journal Article
RAC1B modulates intestinal tumourigenesis via modulation of WNT and EGFR signalling pathways
Gudiño, Victoria, Pohl, Sebastian Öther-Gee, Billard, Caroline V., Cammareri, Patrizia, Bolado, Alfonso, Aitken, Stuart, Stevenson, David, Hall, Adam E., Agostino, Mark, Cassidy, John, Nixon, Colin, von Kriegsheim, Alex, Freile, Paz, Popplewell, Linda, Dickson, George, Murphy, Laura, Wheeler, Ann, Dunlop, Malcolm, Din, Farhat, Strathdee, Douglas, Sansom, Owen J., Myant, Kevin B.
Published in Nature communications (20.04.2021)
Published in Nature communications (20.04.2021)
Get full text
Journal Article
Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation
de Klerk, Eleonora, Venema, Andrea, Anvar, S Yahya, Goeman, Jelle J, Hu, Ouhua, Trollet, Capucine, Dickson, George, den Dunnen, Johan T, van der Maarel, Silvère M, Raz, Vered, 't Hoen, Peter A C
Published in Nucleic acids research (01.10.2012)
Published in Nucleic acids research (01.10.2012)
Get full text
Journal Article
Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice
Lu-Nguyen, Ngoc, Ferry, Arnaud, Schnell, Frederick J, Hanson, Gunnar J, Popplewell, Linda, Dickson, George, Malerba, Alberto
Published in Human molecular genetics (15.09.2019)
Published in Human molecular genetics (15.09.2019)
Get full text
Journal Article
Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease
Harish, Pradeep, Forrest, Leysa, Herath, Shanti, Dickson, George, Malerba, Alberto, Popplewell, Linda
Published in Frontiers in physiology (05.03.2020)
Published in Frontiers in physiology (05.03.2020)
Get full text
Journal Article
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy
Malerba, Alberto, Roth, Fanny, Harish, Pradeep, Dhiab, Jamila, Lu-Nguyen, Ngoc, Cappellari, Ornella, Jarmin, Susan, Mahoudeau, Alexandrine, Ythier, Victor, Lainé, Jeanne, Negroni, Elisa, Abgueguen, Emmanuelle, Simonelig, Martine, Guedat, Philippe, Mouly, Vincent, Butler-Browne, Gillian, Voisset, Cécile, Dickson, George, Trollet, Capucine
Published in Human molecular genetics (15.05.2019)
Published in Human molecular genetics (15.05.2019)
Get full text
Journal Article
Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1
Benlahrech, Adel, Harris, Julian, Meiser, Andrea, Papagatsias, Timos, Hornig, Julia, Hayes, Peter, Lieber, Andre, Athanasopoulos, Takis, Bachy, Veronique, Csomor, Eszter, Daniels, Rod, Fisher, Kerry, Gotch, Frances, Seymour, Len, Logan, Karen, Barbagallo, Romina, Klavinskis, Linda, Dickson, George, Patterson, Steven
Published in Proceedings of the National Academy of Sciences - PNAS (24.11.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (24.11.2009)
Get full text
Journal Article
Langerin negative dendritic cells promote potent CD8⁺ T-cell priming by skin delivery of live adenovirus vaccine microneedle arrays
Bachy, Veronique, Hervouet, Catherine, Becker, Pablo D., Chorro, Laurent, Carlin, Leo M., Herath, Shanthi, Papagatsias, Timos, Barbaroux, Jean-Baptiste, Oh, Sea-Jin, Benlahrech, Adel, Athanasopoulos, Takis, Dickson, George, Patterson, Steven, Kwon, Sung-Yun, Geissmann, Frederic, Klavinskis, Linda S.
Published in Proceedings of the National Academy of Sciences - PNAS (19.02.2013)
Published in Proceedings of the National Academy of Sciences - PNAS (19.02.2013)
Get full text
Journal Article
Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles
Peccate, Cécile, Mollard, Amédée, Le Hir, Maëva, Julien, Laura, McClorey, Graham, Jarmin, Susan, Le Heron, Anita, Dickson, George, Benkhelifa-Ziyyat, Sofia, Piétri-Rouxel, France, Wood, Matthew J, Voit, Thomas, Lorain, Stéphanie
Published in Human molecular genetics (15.08.2016)
Published in Human molecular genetics (15.08.2016)
Get full text
Journal Article
Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)
Harish, Pradeep, Malerba, Alberto, Lu‐Nguyen, Ngoc, Forrest, Leysa, Cappellari, Ornella, Roth, Fanny, Trollet, Capucine, Popplewell, Linda, Dickson, George
Published in Journal of cachexia, sarcopenia and muscle (01.10.2019)
Published in Journal of cachexia, sarcopenia and muscle (01.10.2019)
Get full text
Journal Article