Advances in gene therapy for muscular dystrophies [version 1; referees: 2 approved]

by Hayder Abdul-Razak, Alberto Malerba, George Dickson
Published 2016-08-01

Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit <i>DUX4</i> Expression

by Ngoc Lu-Nguyen, George Dickson, Alberto Malerba, Linda Popplewell
Published 2022-07-01

Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease

by Pradeep Harish, Leysa Forrest, Shanti Herath, George Dickson, Alberto Malerba, Linda Popplewell
Published 2020-03-01

Synapsin I phosphorylation is dysregulated by beta-amyloid oligomers and restored by valproic acid

by Jade Marsh, Saifuddien Haji Bagol, Robin S.B. Williams, George Dickson, Pavlos Alifragis
Published 2017-10-01

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

by Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell, Fred Schnell, Gunnar Hanson, George Dickson
Published 2017-03-01

Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)

by Pradeep Harish, Alberto Malerba, Ngoc Lu‐Nguyen, Leysa Forrest, Ornella Cappellari, Fanny Roth, Capucine Trollet, Linda Popplewell, George Dickson
Published 2019-10-01

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of D...

by Alberto Malerba, Jagjeet K Kang, Graham McClorey, Amer F Saleh, Linda Popplewell, Michael J Gait, Matthew JA Wood, George Dickson
Published 2012-01-01

Fusion of ubiquitin to HIV gag impairs human monocyte-derived dendritic cell maturation and reduces ability to induce gag T cell responses.

by Shanthi Herath, Adel Benlahrech, Timos Papagatsias, Takis Athanasopoulos, Zineb Bouzeboudjen, Catherine Hervouet, Linda Klavinskis, Andrea Meiser, Peter Kelleher, George Dickson, Steven Patterson
Published 2014-01-01

Fragmentation of SIV-gag vaccine induces broader T cell responses.

by Adel Benlahrech, Andrea Meiser, Shanthi Herath, Timos Papagatsias, Takis Athanasopoulos, Fucheng Li, Steve Self, Veronique Bachy, Catherine Hervouet, Karen Logan, Linda Klavinskis, George Dickson, Steven Patterson
Published 2012-01-01

Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

by Vassilia Partali, Michael D. Pungente, Aseel Abu-Dayya, Tushar Khanna, Marcella Flinterman, Nada Abdul Khalique, George Dickson, Liji Raju, Hans-Richard Sliwka, Christer L. Øpstad, Linda J. Popplewell
Published 2012-01-01

Mutation-Directed Therapeutics for Neurofibromatosis Type I

by Andre Leier, David M. Bedwell, Ann T. Chen, George Dickson, Kim M. Keeling, Robert A. Kesterson, Bruce R. Korf, Tatiana T. Marquez Lago, Ulrich F. Müller, Linda Popplewell, Jiangbing Zhou, Deeann Wallis
Published 2020-06-01

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

by Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni
Published 2020-06-01

BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

by Vanessa Strings-Ufombah, Alberto Malerba, Shih-Chu Kao, Sonal Harbaran, Fanny Roth, Ornella Cappellari, Ngoc Lu-Nguyen, Keiko Takahashi, Sophie Mukadam, Georgina Kilfoil, Claudia Kloth, Petrus Roelvink, George Dickson, Capucine Trollet, David Suhy
Published 2021-06-01

Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I

by André Leier, Marc Moore, Hui Liu, Michael Daniel, Alexis M. Hyde, Ludwine Messiaen, Bruce R. Korf, Jamuna Selvakumaran, Lukasz Ciszewski, Laura Lambert, Jeremy Foote, Margaret R. Wallace, Robert A. Kesterson, George Dickson, Linda Popplewell, Deeann Wallis
Published 2022-06-01

Mitochondrial dysfunction reveals the role of mRNA poly(A) tail regulation in oculopharyngeal muscular dystrophy pathogenesis.

by Aymeric Chartier, Pierre Klein, Stéphanie Pierson, Nicolas Barbezier, Teresa Gidaro, François Casas, Steven Carberry, Paul Dowling, Laurie Maynadier, Maëlle Bellec, Martine Oloko, Claude Jardel, Bodo Moritz, George Dickson, Vincent Mouly, Kay Ohlendieck, Gillian Butler-Browne, Capucine Trollet, Martine Simonelig
Published 2015-03-01

Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy

by Sara Benedetti, Narumi Uno, Hidetoshi Hoshiya, Martina Ragazzi, Giulia Ferrari, Yasuhiro Kazuki, Louise Anne Moyle, Rossana Tonlorenzi, Angelo Lombardo, Soraya Chaouch, Vincent Mouly, Marc Moore, Linda Popplewell, Kanako Kazuki, Motonobu Katoh, Luigi Naldini, George Dickson, Graziella Messina, Mitsuo Oshimura, Giulio Cossu, Francesco Saverio Tedesco
Published 2018-02-01

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.

by Monika Hiller, Maria Sofia Falzarano, Iker Garcia-Jimenez, Valentina Sardone, Ruurd C Verheul, Linda Popplewell, Karen Anthony, Estibaliz Ruiz-Del-Yerro, Hana Osman, Jelle J Goeman, Kamel Mamchaoui, George Dickson, Alessandra Ferlini, Francesco Muntoni, Annemieke Aartsma-Rus, Virginia Arechavala-Gomeza, Nicole A Datson, Pietro Spitali
Published 2018-01-01

RAC1B modulates intestinal tumourigenesis via modulation of WNT and EGFR signalling pathways

by Victoria Gudiño, Sebastian Öther-Gee Pohl, Caroline V. Billard, Patrizia Cammareri, Alfonso Bolado, Stuart Aitken, David Stevenson, Adam E. Hall, Mark Agostino, John Cassidy, Colin Nixon, Alex von Kriegsheim, Paz Freile, Linda Popplewell, George Dickson, Laura Murphy, Ann Wheeler, Malcolm Dunlop, Farhat Din, Douglas Strathdee, Owen J. Sansom, Kevin B. Myant
Published 2021-04-01

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

by Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, Taeyoung Koo, Jean-Laurent Thibaut, Béatrice Matot, Marie Devaux, Johanne Le Duff, Jack-Yves Deschamps, Inès Barthelemy, Stéphane Blot, Isabelle Testault, Karim Wahbi, Stéphane Ederhy, Samia Martin, Philippe Veron, Christophe Georger, Takis Athanasopoulos, Carole Masurier, Federico Mingozzi, Pierre Carlier, Bernard Gjata, Jean-Yves Hogrel, Oumeya Adjali, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson
Published 2017-07-01

An analysis of vibration sensitivity in hydrophone design /

by Hugus, George Dickson
Published [198