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

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

Publikováno v: International Journal of Molecular Sciences; Volume 23; Issue 11; Pages: 6298
Digibug. Repositorio Institucional de la Universidad de Granada
instname

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.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d62b42463b12f12e03250746a3f4fc2e