Article
C. elegans expressing D76N β2-microglobulin: a model for in vivo screening of drug candidates targeting amyloidosis.
Scientific reports - 27 Dec 2019
Faravelli Giulia, Raimondi Sara, Marchese Loredana, Partridge Frederick A, Soria Cristina, Mangione P Patrizia, Canetti Diana, Perni Michele, Aprile Francesco A, Zorzoli Irene, Di Schiavi Elia, Lomas David A, Bellotti Vittorio, Sattelle David B, Giorgetti Sofia
Abstract excerpt
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...
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