Can we use fish to model FA?
A tractable vertebrate model that recapitulates key aspects of Friedreich ataxia
Animal models of Friedreich’s ataxia (FA) are important to study the disease and test potential therapies before they are assessed in humans. There are many mouse models of FA that have been created to study different aspects of the disease; however, these models are not perfect.
Dr. Robert Wilson at the Children’s Hospital of Philadelphia has used gene editing to delete the frataxin gene in zebrafish, effectively creating a zebrafish model of FA.
These FA zebrafish rapidly develop a more pronounced phenotype than FA mice, making the testing of potential therapies faster. They are also cheaper to work with and have some features that are more closely related to humans.
We are pleased to award a grant to Dr. Robert Wilson to further explore this FA zebrafish model. FARA is co-funding this grant in partnership with FARA Ireland (https://faraireland.eu/). In this grant, Dr. Wilson’s lab will validate additional characteristics of this model.
Dr. Wilson’s lab will also use this model to try to identify drug effects that may be predictive of efficacy in humans. They will do this by dosing the FA zebrafish with drugs that have not shown efficacy in clinical trials and drugs that have (such as omaveloxolone). The goal is to identify a predictor of drug efficacy that can be used to increase the likelihood that drugs in the clinical trial pipeline will show efficacy in humans.