Cell BiologybioRxivPreprint
Disease-associated programming of cell memory in glycogen storage disorder type 1a
Uri Sprecher, J D’Souza, Kumudesh Mishra…·Miguel Weil
This paper shows that GSD1a patient fibroblasts retain a reversible disease phenotype in vitro, driven by epigenetic and transcriptional changes, and that a drug can reverse these effects.
3 citations