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

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

Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

por Kaiser, Robert A., Weber, Nicholas D., Trigueros‐Motos, Laia, Allen, Kari L., Martinez, Michael, Cao, William, VanLith, Caitlin J., Hillin, Lori G., Douar, Anne, González‐Aseguinolaza, Gloria, Aldabe, Rafael, Lillegard, Joseph B.
Publicado 2021

AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

por Buj-Bello, Anna, Fougerousse, Françoise, Schwab, Yannick, Messaddeq, Nadia, Spehner, Danièle, Pierson, Christopher R., Durand, Muriel, Kretz, Christine, Danos, Olivier, Douar, Anne-Marie, Beggs, Alan H., Schultz, Patrick, Montus, Marie, Denèfle, Patrice, Mandel, Jean-Louis
Publicado 2008

Optogenetic therapy: high spatiotemporal resolution and pattern discrimination compatible with vision restoration in non-human primates

por Gauvain, Gregory, Akolkar, Himanshu, Chaffiol, Antoine, Arcizet, Fabrice, Khoei, Mina A., Desrosiers, Mélissa, Jaillard, Céline, Caplette, Romain, Marre, Olivier, Bertin, Stéphane, Fovet, Claire-Maelle, Demilly, Joanna, Forster, Valérie, Brazhnikova, Elena, Hantraye, Philippe, Pouget, Pierre, Douar, Anne, Pruneau, Didier, Chavas, Joël, Sahel, José-Alain, Dalkara, Deniz, Duebel, Jens, Benosman, Ryad, Picaud, Serge
Publicado 2021