Neural Stem Cell Laboratory
Affiliation and Location
Università degli Studi di Milano
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico
Contacts and References
Head of Laboratory: Prof.ssa Stefania Corti
E-mail: stefania.corti@unimi.it stefania.corti@policlinico.mi.it
Since 2006, the laboratory has contributed to the development of therapeutic strategies for neuromuscular and neurodegenerative diseases.
Key milestones include:
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Stem Cell Research: Developed the first experimental stem cell-based approach for motor neuron diseases, including pediatric-onset conditions—such as Spinal Muscular Atrophy (SMA) and Spinal Muscular Atrophy with Respiratory Distress (SMARD1)—and adult-onset conditions like Amyotrophic Lateral Sclerosis (ALS).
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Gene Therapy: Pioneered gene therapy strategies for SMARD1 and hereditary neuropathies, such as Charcot-Marie-Tooth disease type 2 (CMT2A).
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Antisense Oligonucleotides: Contributed significantly to the advancement of antisense oligonucleotide (ASO) therapies for both SMA and ALS.
The laboratory actively collaborates with the Laboratory of Genetics and Biochemistry, an Italian and international reference center providing high-level molecular diagnostics for neurological and neuromuscular diseases.
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Genomic Analysis: Our services include the analysis of over 100 genes associated with neurological disorders, combined with biochemical and protein-based methodologies.
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Protein Characterization: We perform detailed studies to characterize the muscle proteins involved in these pathologies.
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Personalized Medicine: Understanding the link between genetic variations and clinical presentation is a fundamental step in our mission to develop personalized medicine approaches and targeted treatments.
Our research is dedicated to identifying innovative solutions for neurological diseases, employing two primary approaches: cell therapies using stem cells and molecular therapies (gene therapy and antisense oligonucleotides).
Disease Modeling and Organoids
We have developed sophisticated 2D and 3D laboratory models that replicate patients’ brain, muscle, and spinal cord tissues. These “mini-organs” (organoids) enable us to:
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Understand the pathogenic mechanisms of various disorders.
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Test potential new treatments through a personalized medicine lens.
Our team operates within a multidisciplinary environment, merging diverse expertise to identify novel therapeutic targets and pioneering treatment modalities.
The team
Contact persons
Head of Laboratory: Prof.ssa Stefania Corti
Contacts and informations
E-mail: stefania.corti@unimi.it
stefania.corti@policlinico.mi.it
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