Research
Dr. Iacovino’s lab is dedicated to advancing the understanding and treatment of neurodegenerative and neurodevelopmental disorders through cutting-edge stem cell technologies and translational research. A key focus of their work is disease modeling of Mucopolysaccharidosis type III (MPS III, or Sanfilippo syndrome) using human pluripotent stem cells.
By differentiating these cells into neurons and brain organoids, we aim to unravel disease mechanisms at the cellular and tissue levels, providing critical insights into neurodegeneration in MPS III. In parallel, we are actively developing and evaluating neural progenitor cell therapy as a potential treatment for Sanfilippo syndrome.
This includes ongoing preclinical studies as well as participation in clinical research initiatives, such as an Anakinra clinical trial, to assess anti-inflammatory interventions in affected individuals. Iacovino’s lab also extends its translational focus to Pitt-Hopkins syndrome, a profound form of autism, where we are investigating antisense oligonucleotide (ASO)-based therapeutics to correct gene expression and improve neurological outcomes. Through these interdisciplinary efforts, they strive to bridge the gap between bench and bedside, offering hope for individuals affected by rare pediatric neurological disorders.
Biography
Dr. Michelina Iacovino is an Investigator at The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center and an Associate Professor of Pediatrics at the David Geffen School of Medicine at UCLA. Trained in biochemistry and stem cell biology, she earned her Ph.D. from the University of Molise in Campobasso, Italy, and completed postdoctoral training in stem cell and developmental biology at UT Southwestern Medical Center and the University of Minnesota.
Her research focuses on the development of stem cell-based therapies for rare pediatric genetic diseases, with particular emphasis on Sanfilippo syndrome (MPS III), a devastating lysosomal storage disorder that currently has no effective treatment, and Pitt Hopkins Syndromes, a rare disease in the Autism spectrum.