Genetic and pharmacological inhibition of calcineurin corrects the BDNF transport defect in Huntington's disease.

ABSTRACT:

Background

Huntington's disease (HD) is an inherited neurogenerative disease caused by an abnormal expansion of glutamine repeats in the huntingtin protein. There is currently no treatment to prevent the neurodegeneration caused by this devastating disorder. Huntingtin has been shown to be a positive regulator of vesicular transport, particularly for neurotrophins such as brain-derived neurotrophic factor (BDNF). This function is lost in patients with HD, resulting in a decrease in neurotrophic support and subsequent neuronal death. One promising line of treatment is therefore the restoration of huntingtin function in BDNF transport.

Results

The phosphorylation of huntingtin at serine 421 (S421) restores its function in axonal transport. We therefore investigated whether inh

SUBMITTER: Pineda JR 

PROVIDER: S-EPMC2776580 | biostudies-literature | 2009 Oct

REPOSITORIES: biostudies-literature