Mathilde Cohen-Tannoudji

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

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

Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation

Maria Chatzifrangkeskou, David Yadin, Thibaut Marais et al. · Human Molecular Genetics · 2018 · 64 citations · Full text

Life Sciences, Cardiology, Cell Biology +17

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Pasqualina Colella, Pauline Sellier, Manuel J. Gómez et al. · EBioMedicine · 2020 · 27 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +14

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A

Maria Grazia Biferi, Mathilde Cohen-Tannoudji, Andrea García-Silva et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 27 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +13