Associate Professor

Amelia Morrone, PhD, is an Associate Professor of Paediatrics at the University of Florence and Head of the Molecular Genetics Laboratory of Neurometabolic Diseases at the Meyer Paediatric Hospital, IRCCS, in Florence. She holds a PhD in Neurometabolic Sciences and is board-certified in both Medical Genetics and Biochemical and Clinical Chemistry. She completed her PhD and postdoctoral training in the laboratory of Dr. S. D’Azzo in Rotterdam (Erasmus University, The Netherlands) and Dr. E.P. Hoffman in Pittsburgh (PA, USA), acquiring deep expertise in the molecular and cellular biology of lysosomal enzymes, both in their wild-type and mutant forms. Her research focuses on genetic, molecular, cellular, and functional studies, iPSC reprogramming, and neuronal differentiation for neurometabolic diseases, with particular interest in lysosomal storage diseases (LSDs).
She is involved in collaborative studies on the development of small molecules (pharmacological chaperones) for the potential treatment of LSDs and in epigenetic studies on cobalamin CblC/epi-CblC disease. She is the author or co-author of 167 peer-reviewed publications in international scientific journals (Scopus, September 2025).