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

Aguilar-González, A., González-Correa, J. E., Barriocanal-Casado, E., Ramos-Hernández, I., Lerma-Juárez, M. A., Greco, S., Rodríguez-Sevilla, J. J., Molina-Estévez, F. J., Montalvo-Romeral, V., Ronzitti, G., Sánchez-Martín, R. M., Martín, F., & Muñoz, P. (2022). Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies. International Journal of Molecular Sciences, 23(11), 6298. https://doi.org/10.3390/ijms23116298
Authors:
Araceli Aguilar-Gonzalez
Juan Elias Gonzalez-Correa
Eliana Barriocanal-Casado
Iris Ramos-Hernandez
Sara Greco
Juan Jose Rodriguez-Sevilla
Francisco Javier Molina-Estevez
Rosario Maria Sanchez-Martin
Francisco Martin
Pilar Munoz
Miguel A. Lerma-Juarez
Valle Montalvo-Romeral
Giuseppe Ronzitti
Affiliated Authors:
Eliana Barriocanal-Casado
Author Keywords:
pompe disease
lentiviral vectors
cellular disease models
optimised gaa (acid alpha-glucosidase)
crispr
cas9 technology
adeno-associated virus
Publication Type:
Article
Unique ID:
10.3390/ijms23116298
PMID:
Publication Date:
Data Source:
Web of Science

Record Created: