Ngoc Lu‐Nguyen

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

Taeyoung Koo, Ngoc Lu‐Nguyen, Alberto Malerba et al. · Molecular Therapy · 2018 · 82 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

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

Haiyan Zhou, Jinhong Meng, Alberto Malerba et al. · Journal of Cachexia Sarcopenia and Muscle · 2020 · 75 citations · Full text

Medicine, Neurology, Neuroscience +17

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

Pradeep Harish, Alberto Malerba, Ngoc Lu‐Nguyen et al. · Journal of Cachexia Sarcopenia and Muscle · 2019 · 39 citations · Full text

Medicine, Immunology, Pharmacotherapy +17

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

Ngoc Lu‐Nguyen, Alberto Malerba, Linda Popplewell et al. · Molecular Therapy — Nucleic Acids · 2016 · 37 citations · Full text

Medicine, Molecular Biology, Molecular Medicine +15

Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy

Alberto Malerba, Fanny Roth, Pradeep Harish et al. · Human Molecular Genetics · 2019 · 32 citations · Full text

Life Sciences, Molecular Biology, Molecular Medicine +17