In vivo and in vitro genome editing to explore GNE functions

GNE myopathy is an adult onset neuromuscular disorder characterized by slowly progressive distal and proximal muscle weakness, caused by missense recessive mutations in the GNE gene. Although the encoded bifunctional enzyme is well known as the limiting factor in the biosynthesis of sialic acid, no...

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Detalles Bibliográficos
Autores principales: Ilouz, Nili, Harazi, Avi, Guttman, Miriam, Daya, Alon, Ruppo, Shmuel, Yakovlev, Lena, Mitrani-Rosenbaum, Stella
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Genome Editing
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9552322/
https://www.ncbi.nlm.nih.gov/pubmed/36237634
http://dx.doi.org/10.3389/fgeed.2022.930110

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9552322/
https://www.ncbi.nlm.nih.gov/pubmed/36237634
http://dx.doi.org/10.3389/fgeed.2022.930110

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