Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies

por Aguilar-González, Araceli, González-Correa, Juan Elías, Barriocanal-Casado, Eliana, Ramos-Hernández, Iris, Lerma-Juárez, Miguel A., Greco, Sara, Rodríguez-Sevilla, Juan José, Molina-Estévez, Francisco Javier, Montalvo-Romeral, Valle, Ronzitti, Giuseppe, Sánchez-Martín, Rosario María, Martín, Francisco, Muñoz, Pilar
Publicado 2022

A Comprehensive, Ethnically Diverse Library of Sickle Cell Disease-Specific Induced Pluripotent Stem Cells

por Park, Seonmi, Gianotti-Sommer, Andreia, Molina-Estevez, Francisco Javier, Vanuytsel, Kim, Skvir, Nick, Leung, Amy, Rozelle, Sarah S., Shaikho, Elmutaz Mohammed, Weir, Isabelle, Jiang, Zhihua, Luo, Hong-Yuan, Chui, David H.K., Figueiredo, Maria Stella, Alsultan, Abdulraham, Al-Ali, Amein, Sebastiani, Paola, Steinberg, Martin H., Mostoslavsky, Gustavo, Murphy, George J.
Publicado 2017