Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings

por Chand, Deepa H., Sun, Rui, Diab, Karim A., Kenny, Damien, Tukov, Francis Fonyuy
Publicado 2023

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys

por Tukov, Francis Fonyuy, Mansfield, Keith, Milton, Mark, Meseck, Emily, Penraat, Kelley, Chand, Deepa, Hartmann, Andreas
Publicado 2022

Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates

por Johnson, Eric W., Sutherland, Jeffrey J., Meseck, Emily, McElroy, Cameron, Chand, Deepa H., Tukov, Francis Fonyuy, Hudry, Eloise, Penraat, Kelley
Publicado 2022

Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa

por MacLachlan, Timothy K., Milton, Mark N., Turner, Oliver, Tukov, Francis, Choi, Vivian W., Penraat, Jan, Delmotte, Marie-Hélène, Michaut, Lydia, Jaffee, Bruce D., Bigelow, Chad E.
Publicado 2017

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021