C. elegans expressing D76N β₂-microglobulin: a model for in vivo screening of drug candidates targeting amyloidosis.

ABSTRACT: The availability of a genetic model organism with which to study key molecular events underlying amyloidogenesis is crucial for elucidating the mechanism of the disease and the exploration of new therapeutic avenues. The natural human variant of β₂-microglobulin (D76N β₂-m) is associated with a fatal familial form of systemic amyloidosis. Hitherto, no animal model has been available for studying in vivo the pathogenicity of this protein. We have established a transgenic C. elegans line, expressing the human D76N β₂-m variant. Using the INVertebrate Automated Phenotyping Platform (INVAPP) and the algorithm Paragon, we were able to detect growth and motility impairment in D76N β₂-m expressing worms. We also demonstrated the specificity of the β

SUBMITTER: Faravelli G 

PROVIDER: S-EPMC6934621 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature