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

Genetics
Katherine A StrynatkaChristopher R McMaster

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.

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Citations

May 24, 2018·Diseases·Máté VargaKálmán Tory
Jul 2, 2020·G3 : Genes - Genomes - Genetics·Mitchell A EllisonKaren M Arndt
Dec 15, 2019·Nature Reviews. Drug Discovery·Erik TambuyzerMarco Prunotto
Sep 21, 2018·Frontiers in Genetics·Alberto Rissone, Shawn M Burgess
Jun 3, 2021·Pharmaceuticals·Hung-Chieh LeeHuai-Jen Tsai

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Methods Mentioned

BETA
transgenic
SGA
genetic array technology
GTPase
gene knockdown
dissection
genetic modifications
genotyping
xenograft

Software Mentioned

HIPHOP
MARCH

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