Betul Beyza Gunes

Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich’s ataxia mice

Friedreich’s ataxia (FRDA) is a multisystemic neurodegenerative disorder caused by GAA repeat expansion in intron 1 of frataxin ( FXN ), resulting in FXN deficiency and mitochondrial dysfunction. Current therapies do not correct the underlying genetic defect. Here, we demonstrate...

Selective cGAS Inhibition Is Cardioprotective After Myocardial Infarction

Activation of the DNA sensor cGAS has emerged as a potential driver of maladaptive inflammation after myocardial infarction. In this study, the authors provide evidence that a newly developed pharmacologic cGAS inhibitor improves cardiac recovery following myocardial infarction....