Experimental Neuroscience Research Laboratory
Affiliation and Location
IRCCS Istituto Auxologico Italiano
Università degli Studi di Milano
Centro di Ricerche e Tecnologie Biomediche
Via G. Zucchi 18
Cusano Milanino
Contacts and References
Head of the Laboratory: Prof. Vincenzo Silani
Neurologist, Professor Emerit
E-mail / Ph: vincenzo@silani.com / 02 61911 2982
Since 2002, with the establishment of the “Centro Dino Ferrari ” branch at the IRCCS Istituto Auxologico Italiano, the Experimental Neuroscience Research Laboratory has been dedicated to the study of neuromuscular and neurodegenerative pathologies. Its focus includes genetic diagnosis, the acquisition of biomarkers from cerebrospinal fluid (CSF) and serum, and the study of pathology in vitro using differentiated human cells derived from patient iPSCs to test molecules of potential clinical efficacy.
Over the years, the laboratory has contributed—through constantly updated technologies (NGS)—to the identification of more than 10 pathogenic genes for Amyotrophic Lateral Sclerosis/Frontotemporal Dementia, the discovery of new biomarkers, and the preparation of three-dimensional cellular models (organoids) for a more accurate definition of molecular mechanisms.
In 2025, the “Center for Molecular and Translational Neurobiology” was established on-site; directed by Prof. Antonia Ratti, it is dedicated to defining the molecular mechanisms of various neurodegenerative diseases. The validation of biomarkers in peripheral blood is a preliminary step for the diagnosis of different pathologies in the pre-clinical phase, allowing for the adoption of corrective strategies for pathogenic risks once identified.
The Laboratory offers accurate genetic diagnostics supported by non-genetic biomarkers for various neurodegenerative pathologies, alongside the development of databases aimed at providing the best correlation between genotype and clinical phenotype. Our extensive expertise ensures an up-to-date diagnostic pathway focused on the earliest possible diagnosis of various diseases, enabling personalized and early treatment.
Today, particular attention is paid to the definition of pre-morbid conditions to correct identified risk factors. Furthermore, significant focus is placed on professional and competent genetic counseling, as well as the determination of blood-based pathology biomarkers for future population screening.
Clinical-diagnostic procedures initiate research into the genetic basis of neurodegenerative diseases, defining specific biomarkers and the cellular mechanisms involved, including potential efficacy testing with molecules for clinical use. The main research lines include: the discovery of new mutations, genotype-phenotype correlation, the identification of new causative genes through advanced sequencing techniques, the characterization of known biomarkers—including those in peripheral blood—and the identification of new markers by studying pathogenetic mechanisms in human in vitro cellular models derived from iPSCs.
The Laboratory collaborates with national and international research centers through extensive exchanges within the IRCCS Network of Neuroscience and Neurorehabilitation (RIN) and the European Reference Network for Neuromuscular Diseases (ERN-NMD).