Cysteamine Protects Neurons from Mutant Huntingtin Toxicity.

ABSTRACT:

Background

The potential benefit of cysteamine for Huntington's disease has been demonstrated in HD animal models. Cysteamine and its derivate cystamine were shown to reduce neuropathology and prolong lifespan. Human studies have demonstrated safety, and suggestive results regarding efficacy. Despite all the studies available in vivo, there are only few in vitro studies, and the mechanism of action of cysteamine remains unclear.

Objective

The objective of this study is to assess the capacity of cysteamine for neuroprotection against mutant Huntingtin in vitro using cellular models of HD, and to provide initial data regarding mechanism of action.

Methods

We tested the neuroprotective properties of cysteamine in vitro in our primary neuron and iPSC models of HD.

Resu

SUBMITTER: Arbez N 

PROVIDER: S-EPMC7039181 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature