mRNA and gene editing: Late breaking therapies in liver diseases

por Zabaleta, Nerea, Torella, Laura, Weber, Nicholas D., Gonzalez‐Aseguinolaza, Gloria
Publicado 2022

Novel vectors and approaches for gene therapy in liver diseases

por Maestro, Sheila, Weber, Nicholas D., Zabaleta, Nerea, Aldabe, Rafael, Gonzalez-Aseguinolaza, Gloria
Publicado 2021

Immunogenicity of an AAV-Based COVID-19 Vaccine in Murine Models of Obesity and Aging

por Maciorowski, Dawid, Diop, Cheikh, Bhatt, Urja, Estelien, Reynette, Li, Dan, Chauhan, Ruchi, Vandenberghe, Luk H., Zabaleta, Nerea
Publicado 2022

Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate

por Kumar, Prashant, Wang, Michael, Kumru, Ozan S., Hickey, John M., Sanmiguel, Julio, Zabaleta, Nerea, Vandenberghe, Luk H., Joshi, Sangeeta B., Volkin, David B.
Publicado 2023

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

por 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
Publicado 2020

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

por 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
Publicado 2020

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

por 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.
Publicado 2018

Ancestral library identifies conserved reprogrammable liver motif on AAV capsid

por Zinn, Eric, Unzu, Carmen, Schmit, Pauline F., Turunen, Heikki T., Zabaleta, Nerea, Sanmiguel, Julio, Fieldsend, Allegra, Bhatt, Urja, Diop, Cheikh, Merkel, Erin, Gurrala, Rakesh, Peacker, Bryan, Rios, Christopher, Messemer, Kathleen, Santos, Jennifer, Estelien, Reynette, Andres-Mateos, Eva, Wagers, Amy J., Tipper, Christopher, Vandenberghe, Luk H.
Publicado 2022

Computed tomography and [(18)F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates

por Naninck, Thibaut, Kahlaoui, Nidhal, Lemaitre, Julien, Maisonnasse, Pauline, De Mori, Antoine, Pascal, Quentin, Contreras, Vanessa, Marlin, Romain, Relouzat, Francis, Delache, Benoît, Hérate, Cécile, Aldon, Yoann, van Gils, Marit, Zabaleta, Nerea, Ho Tsong Fang, Raphaël, Bosquet, Nathalie, Sanders, Rogier W., Vandenberghe, Luk H., Chapon, Catherine, Le Grand, Roger
Publicado 2022

Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice

por Reichart, Daniel, Newby, Gregory A., Wakimoto, Hiroko, Lun, Mingyue, Gorham, Joshua M., Curran, Justin J., Raguram, Aditya, DeLaughter, Daniel M., Conner, David A., Marsiglia, Júlia D. C., Kohli, Sajeev, Chmatal, Lukas, Page, David C., Zabaleta, Nerea, Vandenberghe, Luk, Liu, David R., Seidman, Jonathan G., Seidman, Christine
Publicado 2023

Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques

por Zabaleta, Nerea, Bhatt, Urja, Hérate, Cécile, Maisonnasse, Pauline, Sanmiguel, Julio, Diop, Cheikh, Castore, Sofia, Estelien, Reynette, Li, Dan, Dereuddre-Bosquet, Nathalie, Cavarelli, Mariangela, Gallouët, Anne-Sophie, Pascal, Quentin, Naninck, Thibaut, Kahlaoui, Nidhal, Lemaitre, Julien, Relouzat, Francis, Ronzitti, Giuseppe, Thibaut, Hendrik Jan, Montomoli, Emanuele, Wilson, James M., Le Grand, Roger, Vandenberghe, Luk H.
Publicado 2022

An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates

por Zabaleta, Nerea, Dai, Wenlong, Bhatt, Urja, Hérate, Cécile, Maisonnasse, Pauline, Chichester, Jessica A., Sanmiguel, Julio, Estelien, Reynette, Michalson, Kristofer T., Diop, Cheikh, Maciorowski, Dawid, Dereuddre-Bosquet, Nathalie, Cavarelli, Mariangela, Gallouët, Anne-Sophie, Naninck, Thibaut, Kahlaoui, Nidhal, Lemaitre, Julien, 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., Contreras, Vanessa, Marlin, Romain, Tsong Fang, Raphaël Ho, Chapon, Catherine, van der Werf, Sylvie, 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., Le Grand, Roger, Vandenberghe, Luk H.
Publicado 2021