Stéphanie Astord

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Elisa Dominguez, Thibaut Marais, Nicolas Chatauret et al. · Human Molecular Genetics · 2010 · 293 citations · Full text

Medicine, Genetics, Neurology +17

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Francesco Puzzo, Pasqualina Colella, Maria Grazia Biferi et al. · Science Translational Medicine · 2017 · 133 citations

Pharmacy, Biochemistry, Biotechnology +11

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

Sofia Benkhelifa‐Ziyyat, Aurore Besse, Marianne Roda et al. · Molecular Therapy · 2013 · 129 citations · Full text

Pharmacy, Neurobiology, Neuroscience +17

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

Yannick Tanguy, Maria Grazia Biferi, Aurore Besse et al. · Frontiers in Molecular Neuroscience · 2015 · 104 citations · Full text

Medicine, Neurobiology, Neuroscience +17

A rare recessive distal hereditary motor neuropathy with HSJ1 chaperone mutation

Sergiu C. Blumen, Stéphanie Astord, Valérie Robin et al. · Annals of Neurology · 2011 · 72 citations

Medicine, Genetics, Neurology +17