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

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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Bibliographic Details
Main Authors:	Andrés Felipe Leal, Javier Cifuentes, Valentina Quezada, Eliana Benincore-Flórez, Juan Carlos Cruz, Luis Humberto Reyes, Angela Johana Espejo-Mojica, Carlos Javier Alméciga-Díaz
Format:	Article
Language:	English
Published:	MDPI AG 2022-09-01
Series:	International Journal of Molecular Sciences
Subjects:	CRISPR/nCas9 genome editing GM2 gangliosidoses non-viral vectors Sandhoff Tay–Sachs
Online Access:	https://www.mdpi.com/1422-0067/23/18/10672

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