Caenorhabditis elegans for rare disease modeling and drug discovery: strategies and strengths.

ABSTRACT: Although nearly 10% of Americans suffer from a rare disease, clinical progress in individual rare diseases is severely compromised by lack of attention and research resources compared to common diseases. It is thus imperative to investigate these diseases at their most basic level to build a foundation and provide the opportunity for understanding their mechanisms and phenotypes, as well as potential treatments. One strategy for effectively and efficiently studying rare diseases is using genetically tractable organisms to model the disease and learn about the essential cellular processes affected. Beyond investigating dysfunctional cellular processes, modeling rare diseases in simple organisms presents the opportunity to screen for pharmacological or genetic factors capable of ameliorating

SUBMITTER: Kropp PA 

PROVIDER: S-EPMC8380043 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature