Drug targets from genetics: α-synuclein.

ABSTRACT: One of the critical issues in Parkinson disease (PD) research is the identity of the specific toxic, pathogenic moiety. In PD, mutations in α-synuclein (αsyn) or multiplication of the SNCA gene encoding αsyn, result in a phenotype of cellular inclusions, cell death, and brain dysfunction. While the historical point of view has been that the macroscopic aggregates containing αsyn are the toxic species, in the last several years evidence has emerged that suggests instead that smaller soluble species--likely oligomers containing misfolded αsyn--are actually the toxic moiety and that the fibrillar inclusions may even be a cellular detoxification pathway and less harmful. If soluble misfolded species of αsyn are the toxic moieties, then cellular mechanisms that degrade misfolded αsyn would be n

SUBMITTER: Danzer KM 

PROVIDER: S-EPMC3364517 | biostudies-literature | 2011 Sep

REPOSITORIES: biostudies-literature