title: Using Caenorhabditis elegans to study serpinopathies.
abstract: Protein misfolding, polymerization, and/or aggregation are hallmarks of serpinopathies and many other human genetic disorders including Alzheimer's, Huntington's, and Parkinson's disease. While higher organism models have helped shape our understanding of these diseases, simpler model systems, like Caenorhabditis elegans, offer great versatility for elucidating complex genetic mechanisms underlying these diseases. Moreover, recent advances in automated high-throughput methodologies have promoted C. elegans as a useful tool for drug discovery. In this chapter, we describe how one could model serpinopathies in C. elegans and how one could exploit this model to identify small molecule compounds that can be developed into effective therapeutic drugs.
authors.affiliation: Department of Pediatrics, Cell Biology and Physiology, University of Pittsburgh School of Medicine, Children’s Hospital of Pittsburgh of UPMC, Pittsburg, Pennsylvania, USA.
authors.forename: Olivia S
authors.initials: OS
authors.lastname: Long
authors.forename: Sager J
authors.initials: SJ
authors.lastname: Gosai
authors.forename: Joon Hyeok
authors.initials: JH
authors.lastname: Kwak
authors.forename: Dale E
authors.initials: DE
authors.lastname: King
authors.forename: David H
authors.initials: DH
authors.lastname: Perlmutter
authors.forename: Gary A
authors.initials: GA
authors.lastname: Silverman
authors.forename: Stephen C
authors.initials: SC
authors.lastname: Pak
mesh_terms: D000818:Animals; D030801:Animals, Genetically Modified; D017173:Caenorhabditis elegans; D004195:Disease Models, Animal; D015843:Serpins / Q000235:genetics / Q000378:metabolism*
chemical_list: D015843:Serpins