Gene, Stem Cell, and Alternative Therapies for SCA 1

Gene, Stem Cell, and Alternative Therapies for SCA 1

Spinocerebellar ataxia 1 is an autosomal dominant disease characterized by neurodegeneration and motor dysfunction. In disease pathogenesis, polyglutamine expansion within Ataxin-1, a gene involved in transcriptional repression, causes protein nuclear inclusions to form. Most notably, neuronal dysfu...

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Bibliographic Details
Main Authors: Jacob Lewis Wagner, Deirdre Margaret O'Connor, Anthony Donsante, Nicholaus M Boulis
Format: Article
Language:English
Published: Frontiers Media S.A. 2016-08-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Gene Therapy
RNAi
mouse model
Stem Cell Therapy
Ataxin-1
SCA 1
Online Access:http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00067/full

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