How Surrogate and Chemical Genetics in Model Organisms Can Suggest Therapies for Human Genetic Diseases. Issue 3 (1st March 2018)

• Record Type: Journal Article
• Authors: Strynatka, Katherine A; Gurrola-Gal, Michelle C; Berman, Jason N; McMaster, Christopher R
• Is Part Of: Genetics. Volume 208:Issue 3(2018)
• Page Start: 833
• Page End: 851
• Publication Date: 2018-03-01
• Subjects: yeast -- zebrafish -- model organism -- genetic disease -- orphan disease -- cancer -- chemical genetics -- drug discovery; Genetics -- Periodicals; 576.5
• DOI: 10.1534/genetics.117.300124
• Languages: English
• ISSNs: 0016-6731

Abstract

Abstract: Genetic diseases are both inherited and acquired. Many genetic diseases fall under the paradigm of orphan diseases, a disease found in < 1 in 2000 persons. With rapid and cost-effective genome sequencing becoming the norm, many causal mutations for genetic diseases are being rapidly determined. In this regard, model organisms are playing an important role in validating if specific mutations identified in patients drive the observed phenotype. An emerging challenge for model organism researchers is the application of genetic and chemical genetic platforms to discover drug targets and drugs/drug-like molecules for potential treatment options for patients with genetic disease. This review provides an overview of how model organisms have contributed to our understanding of genetic disease, with a focus on the roles of yeast and zebrafish in gene discovery and the identification of compounds that could potentially treat human genetic diseases.