POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches

POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches

Leukodystrophies are a class of rare inherited central nervous system (CNS) disorders that affect the white matter of the brain, typically leading to progressive neurodegeneration and early death. Hypomyelinating leukodystrophies are characterized by the abnormal formation of the myelin sheath durin...

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Bibliographic Details
Main Authors: Stefanie Perrier, Mackenzie A. Michell-Robinson, Geneviève Bernard
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-01-01
Series:Frontiers in Cellular Neuroscience
Subjects:
POLR3-related leukodystrophy
4H leukodystrophy
hypomyelination
gene therapy
gene editing
cell therapy
Online Access:https://www.frontiersin.org/articles/10.3389/fncel.2020.631802/full

Internet

https://www.frontiersin.org/articles/10.3389/fncel.2020.631802/full

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