Ana Buj‐Bello

Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

Virginie Mariot, Romain Joubert, Christophe Hourdé et al. · Nature Communications · 2017 · 137 citations · Full text

Medicine, Neurology, Pharmacotherapy +17

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

Perry B. Shieh, Nancy L. Kuntz, Wolfgang Müller‐Felber et al. · The Lancet Neurology · 2023 · 120 citations · Full text

Medicine, Molecular Medicine, Pharmacotherapy +15

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

David L. Mack, Karine Poulard, Melissa Goddard et al. · Molecular Therapy · 2017 · 109 citations · Full text

Medicine, Immunology, Molecular Medicine +17

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

Jihad El Andari, Edith Renaud-Gabardos, Warut Tulalamba et al. · Science Advances · 2022 · 92 citations · Full text

Pharmacy, Bioengineering, Biotechnology +13

Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice

Mirella Lo Scrudato, Karine Poulard, Célia Sourd et al. · Molecular Therapy · 2019 · 57 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17