TY  - JOUR
AU  - Aguilar-González, Araceli
AU  - González-Correa, Juan Elías
AU  - Barriocanal-Casado, Eliana
AU  - Ramos-Hernández, Iris
AU  - Lerma-Juárez, Miguel A
AU  - Greco, Sara
AU  - Rodríguez-Sevilla, Juan José
AU  - Molina-Estévez, Francisco Javier
AU  - Montalvo-Romeral, Valle
AU  - Ronzitti, Giuseppe
AU  - Sánchez-Martín, Rosario María
AU  - Martín, Francisco
AU  - Muñoz, Pilar
PY  - 2022
DO  - 10.3390/ijms23116298
UR  - http://hdl.handle.net/10668/21164
T2  - International journal of molecular sciences
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  - en
KW  - CRISPR/Cas9 technology
KW  - Pompe disease
KW  - adeno-associated virus
KW  - cellular disease models
KW  - lentiviral vectors
KW  - optimised GAA (acid alpha-glucosidase)
KW  - Animals
KW  - Cell Line
KW  - Dependovirus
KW  - Disease Models, Animal
KW  - Genetic Therapy
KW  - Genetic Vectors
KW  - Glycogen Storage Disease Type II
KW  - Humans
KW  - Mice
KW  - Mice, Knockout
KW  - Muscle Cells
KW  - Muscle, Skeletal
KW  - Mutation
KW  - alpha-Glucosidases
TI  - Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies.
TY  - research article
VL  - 23
ER  -