TY  - GEN 
AU  - Aguilar González, Araceli
AU  - González Correa, Juan Elías
AU  - Barriocanal Casado, Eliana
AU  - Ramos Hernández, Iris
AU  - Greco, Sara
AU  - Rodríguez Sevilla, Juan José
AU  - Molina Estévez, Francisco Javier
AU  - Sánchez Martín, Rosario María
AU  - Martín Molina, Francisco
AU  - Muñoz Fernández, Pilar
PY  - 2022
UR  - http://hdl.handle.net/10481/75770
AB  - 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....
LA  - eng
PB  - MDPI
KW  - Pompe disease
KW  - Lentiviral vectors
KW  - Cellular disease models
KW  - Optimised GAA (acid alphaglucosidase)
KW  - CRISPR/Cas9 technology
KW  - Adeno-associated virus
TI  - Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies
DO  - 10.3390/ijms23116298
ER  -