Article

Inducible and reversible phenotypes in a novel mouse model of Friedreich’s Ataxia

2017-05-12

Abstract excerpt

<h4>ABSTRACT</h4> Friedreich’s ataxia (FRDA), the most common inherited ataxia, is caused by recessive mutations that reduce the levels of frataxin (FXN), a mitochondrial iron binding protein. We developed an inducible mouse model of Fxn deficiency that enabled us to control the onset and progression of disease phenotypes by the modulation of Fxn levels. Systemic knockdown of Fxn in adult mice led to multiple...

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Literature Corpus work: b0c01fce-b889-5189-ba8b-defa09e3a101
DOI: 10.1101/137265

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