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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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מידע ביבליוגרפי
Principais autores: Nili Ilouz, Avi Harazi, Miriam Guttman, Alon Daya, Shmuel Ruppo, Lena Yakovlev, Stella Mitrani-Rosenbaum
פורמט: Artigo
שפה:Inglês
יצא לאור: Frontiers Media S.A. 2022-09-01
סדרה:Frontiers in Genome Editing
נושאים:
גישה מקוונת:https://www.frontiersin.org/articles/10.3389/fgeed.2022.930110/full
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