Alberto Malerba

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Caroline Le Guiner, Laurent Servais, Marie Montus et al. · Nature Communications · 2017 · 230 citations · Full text

Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

Alberto Malerba, Paul Sharp, Ian R. Graham et al. · Molecular Therapy · 2010 · 94 citations · Full text

PABPN1 gene therapy for oculopharyngeal muscular dystrophy

Alberto Malerba, Pierre Klein, Houria Bachtarzi et al. · Nature Communications · 2017 · 92 citations · Full text

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 · 78 citations · Full text

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 · 73 citations · Full text