Novel vectors and approaches for gene therapy in liver diseases

nork Maestro, Sheila, Weber, Nicholas D., Zabaleta, Nerea, Aldabe, Rafael, Gonzalez-Aseguinolaza, Gloria
Argitaratua izan da JHEP Rep (2021)

Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5(ch) and AAV1

nork Majowicz, Anna, Salas, David, Zabaleta, Nerea, Rodríguez-Garcia, Estefania, González-Aseguinolaza, Gloria, Petry, Harald, Ferreira, Valerie
Argitaratua izan da Mol Ther (2017)

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

nork Salas, David, Kwikkers, Karin L., Zabaleta, Nerea, Bazo, Andrea, Petry, Harald, van Deventer, Sander J., Aseguinolaza, Gloria Gonzalez, Ferreira, Valerie
Argitaratua izan da Blood Adv (2019)

Inhibition of MicroRNA 6937 Delays Photoreceptor and Vision Loss in a Mouse Model of Retinitis Pigmentosa

nork Anasagasti, Ander, Lara-López, Araceli, Milla-Navarro, Santiago, Escudero-Arrarás, Leire, Rodríguez-Hidalgo, María, Zabaleta, Nerea, González Aseguinolaza, Gloria, de la Villa, Pedro, Ruiz-Ederra, Javier
Argitaratua izan da Pharmaceutics (2020)

GPR108 Is a Highly Conserved AAV Entry Factor

nork Dudek, Amanda M., Zabaleta, Nerea, Zinn, Eric, Pillay, Sirika, Zengel, James, Porter, Caryn, Franceschini, Jennifer Santos, Estelien, Reynette, Carette, Jan E., Zhou, Guo Ling, Vandenberghe, Luk H.
Argitaratua izan da Mol Ther (2020)

In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

nork López-Manzaneda, Sergio, Ojeda-Pérez, Isabel, Zabaleta, Nerea, García-Torralba, Aída, Alberquilla, Omaira, Torres, Raúl, Sánchez-Domínguez, Rebeca, Torella, Laura, Olivier, Emmanuel, Mountford, Joanne, Ramírez, Juan C., Bueren, Juan A., González-Aseguinolaza, Gloria, Segovia, Jose-Carlos
Argitaratua izan da Mol Ther Methods Clin Dev (2020)

CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

nork Zabaleta, Nerea, Barberia, Miren, Martin-Higueras, Cristina, Zapata-Linares, Natalia, Betancor, Isabel, Rodriguez, Saray, Martinez-Turrillas, Rebeca, Torella, Laura, Vales, Africa, Olagüe, Cristina, Vilas-Zornoza, Amaia, Castro-Labrador, Laura, Lara-Astiaso, David, Prosper, Felipe, Salido, Eduardo, Gonzalez-Aseguinolaza, Gloria, Rodriguez-Madoz, Juan R.
Argitaratua izan da Nat Commun (2018)

Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mice and non-human primates

nork Zabaleta, Nerea, Dai, Wenlong, Bhatt, Urja, Chichester, Jessica A., Sanmiguel, Julio, Estelien, Reynette, Michalson, Kristofer T., Diop, Cheikh, Maciorowski, Dawid, Qi, Wenbin, Hudspeth, Elissa, Cucalon, Allison, Dyer, Cecilia D., Pampena, M. Betina, Knox, James J., LaRocque, Regina C., Charles, Richelle C., Li, Dan, Kim, Maya, Sheridan, Abigail, Storm, Nadia, Johnson, Rebecca I., Feldman, Jared, Hauser, Blake M., Zinn, Eric, Ryan, Aisling, Kobayashi, Dione T., Chauhan, Ruchi, McGlynn, Marion, Ryan, Edward T., Schmidt, Aaron G., Price, Brian, Honko, Anna, Griffiths, Anthony, Yaghmour, Sam, Hodge, Robert, Betts, Michael R., Freeman, Mason W., Wilson, James M., Vandenberghe, Luk H.
Argitaratua izan da bioRxiv (2021)