Targeted Antisense Oligonucleotide-Mediated Skipping of Murine <i>Postn</i> Exon 17 Partially Addresses Fibrosis in D2.<i>mdx</i> Mice

by Jessica Trundle, Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell
Published 2024-06-01

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

Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy

by Jinhong Meng, Marc Moore, John Counsell, Francesco Muntoni, Linda Popplewell, Jennifer Morgan
Published 2022-06-01

Expression of the Pro-Fibrotic Marker Periostin in a Mouse Model of Duchenne Muscular Dystrophy

by Jessica Trundle, Viktorija Cernisova, Alexis Boulinguiez, Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell
Published 2024-01-01

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

Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

by Viktorija Cernisova, Ngoc Lu-Nguyen, Jessica Trundle, Shan Herath, Alberto Malerba, Linda Popplewell
Published 2023-05-01

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

Restoration of Normal NF1 Function with Antisense Morpholino Treatment of Recurrent Pathogenic Patient-Specific Variant c.1466A>G; p.Y489C

by Elias K. Awad, Marc Moore, Hui Liu, Lukasz Ciszewski, Laura Lambert, Bruce R. Korf, Linda Popplewell, Robert A. Kesterson, Deeann Wallis
Published 2021-12-01

Novel Metabolomic Approach for Identifying Pathology-Specific Biomarkers in Rare Diseases: A Case Study in Oculopharyngeal Muscular Dystrophy (OPMD)

by Pradeep Harish, Alberto Malerba, Rosemarie H. M. J. M. Kroon, Milad Shademan, Baziel van Engelan, Vered Raz, Linda Popplewell, Stuart G. Snowden
Published 2023-06-01

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

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

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

by Dominic Scaglioni, Francesco Catapano, Matthew Ellis, Silvia Torelli, Darren Chambers, Lucy Feng, Matthew Beck, Caroline Sewry, Mauro Monforte, Shawn Harriman, Erica Koenig, Jyoti Malhotra, Linda Popplewell, Michela Guglieri, Volker Straub, Eugenio Mercuri, Laurent Servais, Rahul Phadke, Jennifer Morgan, Francesco Muntoni
Published 2021-01-01

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