Rate My Professor Rocco Gogliotti

The Gogliotti lab pioneers a translational drug discovery strategy for neurodevelopmental disorders, with a primary focus on Rett syndrome, a rare autism-associated condition stemming from MECP2 gene mutations. By leveraging over 45 human autopsy brain samples—the largest cohort available for Rett syndrome therapeutics—the lab integrates pharmacology, genomics, and neuroscience to pinpoint patient-specific molecular disruptions and validate targets early in development. Central efforts target muscarinic acetylcholine receptors (M1 and M4 subtypes) as therapeutics, demonstrating efficacy in mouse models of Rett syndrome, CDKL5 deficiency disorder, and Pitt Hopkins syndrome; precision medicine addressing mutation-specific gene expression; and RNA-based approaches for MECP2 restoration. This work has attracted major funding, including an R01 grant from the National Institutes of Health, an R21 from the National Institute of Child Health and Human Development, and innovation awards from the International Rett Syndrome Foundation. Notable publications encompass “mGlu7 potentiation rescues cognitive, social, and respiratory phenotypes in a mouse model of Rett syndrome” (Science Translational Medicine, 2017), “Total RNA Sequencing of Rett Syndrome Autopsy Samples Identifies the M4 Muscarinic Receptor as a Novel Therapeutic Target” (Journal of Pharmacology and Experimental Therapeutics, 2018), “mGlu5 positive allosteric modulation normalizes synaptic plasticity defects and motor phenotypes in a mouse model of Rett syndrome” (Human Molecular Genetics, 2016), and “A Coordinated Attack: Rett Syndrome Therapeutic Development” (Trends in Pharmacological Sciences, 2019). Gogliotti earned Loyola’s 2021 Junior Scientist of the Year Award and received a 2025 nomination for Faculty of the Year. He teaches in the Graduate School’s biomedical science programs and mentors students and postdocs in genetics, pharmacology, molecular biology, and neuroscience.