Gene Suppression Therapies in Hereditary Cerebellar Ataxias: A Systematic Review of Animal Studies

Gene Suppression Therapies in Hereditary Cerebellar Ataxias: A Systematic Review of Animal Studies

Introduction: Hereditary cerebellar ataxias (HCAs) are a heterogenous group of neurodegenerative disorders associated with severe disability. Treatment options are limited and overall restricted to symptomatic approaches, leading to poor prognoses. In recent years, there has been extensive research...

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Bibliographic Details
Main Authors: Carolina Santos, Sofia Malheiro, Manuel Correia, Joana Damásio
Format: Article
Language:English
Published: MDPI AG 2023-03-01
Series:Cells
Subjects:
hereditary spinocerebellar degenerations
Machado–Joseph disease
gene silencing
RNA interference
Online Access:https://www.mdpi.com/2073-4409/12/7/1037

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