Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles

Mucopolysaccharidosis IV A (MPS IVA) is a lysosomal disorder caused by mutations in the GALNS gene. Consequently, the glycosaminoglycans (GAGs) keratan sulfate and chondroitin 6-sulfate accumulate in the lysosomal lumen. Although enzyme replacement therapy has shown essential advantages for the pati...

Descripción completa

Detalles Bibliográficos
Autores principales: Leal, Andrés Felipe, Cifuentes, Javier, Torres, Carlos Emilio, Suárez, Diego, Quezada, Valentina, Gómez, Saúl Camilo, Cruz, Juan C., Reyes, Luis H., Espejo-Mojica, Angela Johana, Alméciga-Díaz, Carlos Javier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9440901/
https://www.ncbi.nlm.nih.gov/pubmed/36057729
http://dx.doi.org/10.1038/s41598-022-19407-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9440901/
https://www.ncbi.nlm.nih.gov/pubmed/36057729
http://dx.doi.org/10.1038/s41598-022-19407-x

Ejemplares similares