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

ABSTRACT:

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 on gene suppression therapies (GSTs) as a new hope for disease-modifying strategies. In this article, we aim to perform a review of in vivo studies investigating the efficacy and safety profile of GSTs in HCAs.

Methods

A structured PubMed^® search on GSTs in HCAs from January 1993 up to October 2020 was performed. Inclusion and exclusion criteria were defined, and the selection process was conducted accordingly. The screening process was independently carried out b

SUBMITTER: Santos C 

PROVIDER: S-EPMC10093402 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature