<i>In vivo</i> Functional Consequences of Human <i>THRA</i> Variants Expressed in the Zebrafish

Abstract

Injection of human THRA variants in zebrafish embryos causes tissue-specific defects recapitulating most of the RTHα clinical and biochemical manifestations. The described manipulation of zebrafish embryos represents a novel in vivo model to screen the functional consequences of THRA variants and the rescue potential of new therapeutic compounds.