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Genetics. 2018 Mar;208(3):833-851. doi: 10.1534/genetics.117.300124.

How Surrogate and Chemical Genetics in Model Organisms Can Suggest Therapies for Human Genetic Diseases.

Author information

1
Department of Biochemistry and Molecular Biology, Dalhousie University, Halifax, Nova Scotia, Canada B3H 4R2.
2
Department of Pediatrics, Dalhousie University, Halifax, Nova Scotia, Canada B3K 6R8.
3
Department of Pediatrics, Dalhousie University, Halifax, Nova Scotia, Canada B3K 6R8 jason.berman@iwk.nshealth.ca christopher.mcmaster@dal.ca.
4
Department of Pharmacology, Dalhousie University, Halifax, Nova Scotia, Canada B3H 4R2 jason.berman@iwk.nshealth.ca christopher.mcmaster@dal.ca.

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.

KEYWORDS:

cancer; chemical genetics; drug discovery; genetic disease; model organism; orphan disease; yeast; zebrafish

PMID:
29487144
PMCID:
PMC5844338
DOI:
10.1534/genetics.117.300124
[Indexed for MEDLINE]
Free PMC Article

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