Jean Philippe Combal

Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy

Catherine Vignal, Scott Uretsky, Serge Fitoussi et al. · Ophthalmology · 2018 · 95 citations · Full text

Chemical modification of the adeno-associated virus capsid to improve gene delivery

Mathieu Mével, Mohammed Bouzelha, Aurélien Leray et al. · Chemical Science · 2019 · 87 citations · Full text

Liver Expression of a MiniATP7B Gene Results in Long‐Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice

Oihana Murillo, Daniel Moreno, Cristina Gázquez et al. · Hepatology · 2019 · 50 citations

Optimising the IgG‐degrading enzyme treatment regimen for enhanced adeno‐associated virus transduction in the presence of neutralising antibodies

Irene Ros‐Gañán, Mirja Hommel, Laia Trigueros‐Motos et al. · Clinical & Translational Immunology · 2022 · 30 citations · Full text

High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease

Oihana Murillo, María Collantes, Cristina Gázquez et al. · Molecular Therapy — Methods & Clinical Development · 2022 · 21 citations · Full text