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

Pompe disease (PD) is a rare disorder caused by mutations in the acid alpha-glucosidase (GAA) gene. Most gene therapies (GT) partially rely on the cross-correction of unmodified cells through the uptake of the GAA enzyme secreted by corrected cells. In the present study, we generated isogenic murine...

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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9181599/ https://www.ncbi.nlm.nih.gov/pubmed/35682977 http://dx.doi.org/10.3390/ijms23116298

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