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

Genetics - 1 Mar 2018

Strynatka Katherine A, Gurrola-Gal Michelle C, Berman Jason N, McMaster Christopher R

Abstract excerpt

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...

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Biomarkers
Disease Models, Animal
Genetic Association Studies
Genetic Diseases, Inborn
Neoplasms

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PMID: 29487144
DOI: 10.1534/genetics.117.300124
PMCID: PMC5844338

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