Luoni Mirko
- Details
- Hits: 284
Research scientist
Via Olgettina 58
20132 Milano
Tel 02-26434616
Fax 02-7490574
This email address is being protected from spambots. You need JavaScript enabled to view it.
Gene therapy represents more than just a hope for monogenic neurological diseases. Thanks to this approach, a disease previously considered incurable, such as SMA, has now been treated with astonishing results. Much of this success is strictly linked to the development of Adeno-Associated Viral (AAV) vectors. These vectors currently represent the technology of choice for delivering genetic elements to the nervous system.
Gene therapy based on the use of AAV vectors is my main research interest. During my PhD in Dr. Broccoli's lab, I had the opportunity to work on a new gene therapy approach for Rett syndrome, a devastating neurological disease. In the following years, I contributed to the generation of new AAV variants with increased brain tropism, which could help expand the possibilities of gene therapy.
Today, much of my scientific work is still based on the use of AAVs to optimize new therapeutic approaches for diseases of the nervous system. In parallel, I continue to study Rett syndrome with the aim of understanding the molecular mechanisms behind such a terrible yet fascinating disease.
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.