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CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors

The gangliosidoses GM2 are a group of pathologies mainly affecting the central nervous system due to the impaired GM2 ganglioside degradation inside the lysosome. Under physiological conditions, GM2 ganglioside is catabolized by the β-hexosaminidase A in a GM2 activator protein-dependent mechanism....

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Detalles Bibliográficos
Autores principales:	Leal, Andrés Felipe, Cifuentes, Javier, Quezada, Valentina, Benincore-Flórez, Eliana, Cruz, Juan Carlos, Reyes, Luis Humberto, Espejo-Mojica, Angela Johana, Alméciga-Díaz, Carlos Javier
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9505638/ https://www.ncbi.nlm.nih.gov/pubmed/36142595 http://dx.doi.org/10.3390/ijms231810672

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