Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

por Weber, Nicholas D., Odriozola, Leticia, Martínez-García, Javier, Ferrer, Veronica, Douar, Anne, Bénichou, Bernard, González-Aseguinolaza, Gloria, Smerdou, Cristian
Publicado 2019

Developmentally regulated Tcf7l2 splice variants mediate transcriptional repressor functions during eye formation

por Young, Rodrigo M, Ewan, Kenneth B, Ferrer, Veronica P, Allende, Miguel L, Godovac-Zimmermann, Jasminka, Dale, Trevor C, Wilson, Stephen W
Publicado 2019

Lentiviral vectors can be used for full-length dystrophin gene therapy

por Counsell, John R., Asgarian, Zeinab, Meng, Jinhong, Ferrer, Veronica, Vink, Conrad A., Howe, Steven J., Waddington, Simon N., Thrasher, Adrian J., Muntoni, Francesco, Morgan, Jennifer E., Danos, Olivier
Publicado 2017

Lentiviral vectors can be used for full-length dystrophin gene therapy

por Counsell, John R., Asgarian, Zeinab, Meng, Jinhong, Ferrer, Veronica, Vink, Conrad A., Howe, Steven J., Waddington, Simon N., Thrasher, Adrian J., Muntoni, Francesco, Morgan, Jennifer E., Danos, Olivier
Publicado 2017

Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy

por Counsell, John R., Asgarian, Zeinab, Meng, Jinhong, Ferrer, Veronica, Vink, Conrad A., Howe, Steven J., Waddington, Simon N., Thrasher, Adrian J., Muntoni, Francesco, Morgan, Jennifer E., Danos, Olivier
Publicado 2017

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

por Ros‐Gañán, Irene, Hommel, Mirja, Trigueros‐Motos, Laia, Tamarit, Blanche, Rodríguez‐García, Estefanía, Salas, David, Pérez, Guiomar, Douar, Anne, Combal, Jean Philippe, Benichou, Bernard, Ferrer, Veronica, González‐Aseguinolaza, Gloria
Publicado 2022

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

por Murillo, Oihana, Collantes, Maria, Gazquez, Cristina, Moreno, Daniel, Hernandez-Alcoceba, Ruben, Barberia, Miren, Ecay, Margarita, Tamarit, Blanche, Douar, Anne, Ferrer, Veronica, Combal, Jean Philippe, Peñuelas, Ivan, Bénichou, Bernard, Gonzalez-Aseguinolaza, Gloria
Publicado 2022