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Luoni Mirko

LuoniResearch scientist
c/o Università di Milano - Bicocca
Via Raoul Follereau, 3
20854 Vedano al Lambro (MB)
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Neurogenetics & Genome Editing Lab
The Neurogenetics and Genome Editing Lab focuses on the study of neurodevelopmental disorders and the develoment of innovative gene therapy strategies based on AAV-mediated delivery.
During my training in Dr. Broccoli’s Lab at San Raffaele Hospital, I have primarily focused on Rett syndrome, which remains the central research area of the group. Our work aims to dissect the molecular and functional roles of MECP2, the gene causally mutated in the disease, while simultaneously developing and optimizing novel gene therapy approaches to restore its function or modulate its activity.
This dual strategy, which combines mechanistic understanding with therapeutic development, represents the core framework of our research program and is now being extended to additional severe neurodevelopmental disorders. Building on this framework, I aim to further expand both the mechanistic and therapeutic repertoire of the lab by integrating CRISPR-based genome editing approaches alongside AAV-mediated strategies, with the goal of enabling more precise and effective therapeutic interventions for these severe pathological conditions.

Representative publications
Rossi G, Ordazzo G, Vanni NN, Castoldi V, Iannielli A, Di Silvestre D, Bellini E, Bernardo L, Giannelli SG, Luoni M, Muggeo S, Leocani L, Mauri P, Broccoli V; MCT1-dependent energetic failure and neuroinflammation underlie optic nerve degeneration in Wolfram syndrome mice.  Elife (2023); doi: 10.7554/eLife.81779.

Iannielli A*, Luoni M*, Giannelli SG*, Ferese R, Ordazzo G, Fossati M, Raimondi A, Opazo F, Corti O, Prehn J, Gambardella S, Melki R, Broccoli V. Modeling native and seeded Synuclein aggregation and related cellular dysfunctions in dopaminergic neurons derived by a new set of isogenic iPSC lines with SNCA multiplications. Cell Death Dis. (2022); doi: 10.1038/s41419-022-05330-6

Gigliucci V, Teutsch J, Woodbury-Smith M, Luoni M, Busnelli M, Chini B, A Banerjee A;  Region-Specific KCC2 Rescue by rhIGF-1 and Oxytocin in a Mouse Model of Rett Syndrome. Cerebral Cortex (2021); doi: 10.1093/cercor/bhab388

Luoni M, Giannelli SG, Indrigo M, Niro A, Massimino L, Iannielli A, Passeri L, Russo F, Morabito G, Calamita P, Gregori S, Deverman B, Broccoli V. Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome. Elife (2020); doi: 10.7554/eLife.52629

Rubio A*, Luoni M*, Giannelli SG*, Radice I, Iannielli A, Cancellieri C, Di Berardino C, Regalia G, Lazzari G, Menegon A, Taverna S, Broccoli V. Rapid and efficient CRISPR/Cas9 gene inactivation in human neurons during human pluripotent stem cell differentiation and direct reprogramming. Scientific Reports (2016); doi: 10.1038/srep37540.