Broadening the therapeutic scope for rapamycin treatment.

Abstract:

:The role of autophagy in the degradation of aggregate-prone proteins has been well established. As a result, autophagy upregulation has become an attractive therapeutic strategy for the treatment of proteinopathies, a group of diseases caused by the accumulation of mutant misfolded proteins. We have previously shown that rapamycin attenuates the phenotype in a mouse model of Huntington disease when administered pre-symptomatically and have recently extended this to demonstrate the effectiveness of rapamycin in a transgenic mouse model of spinocerebellar ataxia type 3, a polyglutamine disorder caused by mutations in the ataxin-3 gene. Rapamycin, administered from the initial onset of disease signs, improves motor coordination and results in a decrease in the levels of soluble mutant ataxin-3 and protein aggregates in the brain.

journal_name

Autophagy

journal_title

Autophagy

authors

Menzies FM,Rubinsztein DC

doi

10.4161/auto.6.2.11078

subject

Has Abstract

pub_date

2010-02-01 00:00:00

pages

286-7

issue

2

eissn

1554-8627

issn

1554-8635

pii

11078

journal_volume

6

pub_type

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