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

por Lu-Nguyen, Ngoc, Dickson, George, Malerba, Alberto, Popplewell, Linda
Publicado 2022

Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model

por Lu-Nguyen, Ngoc, Malerba, Alberto, Herath, Shan, Dickson, George, Popplewell, Linda
Publicado 2021

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

por Cernisova, Viktorija, Lu-Nguyen, Ngoc, Trundle, Jessica, Herath, Shan, Malerba, Alberto, Popplewell, Linda
Publicado 2023

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

por Lu-Nguyen, Ngoc, Malerba, Alberto, Popplewell, Linda, Schnell, Fred, Hanson, Gunnar, Dickson, George
Publicado 2017

Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides

por March, James T., Golshirazi, Golnoush, Cernisova, Viktorija, Carr, Heidi, Leong, Yee, Lu-Nguyen, Ngoc, Popplewell, Linda J.
Publicado 2018

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

por Selvakumaran, Jamuna, Ursu, Simona, Bowerman, Melissa, Lu-Nguyen, Ngoc, Wood, Matthew J., Malerba, Alberto, Yáñez-Muñoz, Rafael J.
Publicado 2023

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

por Harish, Pradeep, Malerba, Alberto, Lu‐Nguyen, Ngoc, Forrest, Leysa, Cappellari, Ornella, Roth, Fanny, Trollet, Capucine, Popplewell, Linda, Dickson, George
Publicado 2019

G-quadruplex ligands mediate downregulation of DUX4 expression

por Ciszewski, Lukasz, Lu-Nguyen, Ngoc, Slater, Alex, Brennan, Andrew, Williams, Huw E L, Dickson, George, Searle, Mark S, Popplewell, Linda
Publicado 2020

Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

por Koo, Taeyoung, Lu-Nguyen, Ngoc B., Malerba, Alberto, Kim, Eunji, Kim, Daesik, Cappellari, Ornella, Cho, Hee-Yeon, Dickson, George, Popplewell, Linda, Kim, Jin-Soo
Publicado 2018

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

por 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
Publicado 2020

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

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