Ana Buj Bello is research director at INSERM and head of the Neuromuscular Diseases and GeneTherapy team at Genethon, France. She received her degree in Medicine and Surgery from the University of Lleida, Spain, and a PhD in Neurosciences from the University of St Andrews, UK. She also obtained a diploma in Myology from the University of Pierre et Marie Curie, France. Having done a post-doctorate at the « Institut de Génétique et Biologie Moléculaire et Cellulaire » (IGBMC), France, she joined the INSERM in 2004 and has worked at Genethon since 2009. Her research activities focus on developing AAV-based gene therapies for neuromuscular disorders, in particular congenital myopathies, with a major interest in clinical translation. Her pioneering work on gene replacement therapy for myotubular myopathy has led to the initiation of a clinical trial in patients.