CORTI LAB
About the lab
Corti Lab (CORTILAB) is the research laboratory directed by Prof. Stefania Corti at the Dino Ferrari Center and the University of Milan (UNIMI). We are based in the Sforza and Invernizzi buildings of the Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico in Milan, where basic scientists and clinicians work side by side.
Using patient-derived stem cells, brain and spinal cord organoids and animal models, we study the mechanisms of motor neuron and neuromuscular diseases such as SMA, ALS and CMT2A, and develop cell, gene and RNA-based therapies designed to reach patients.

Research focus
We develop cellular and molecular therapies for neurodegenerative diseases: neural stem cell transplantation, gene therapy and antisense oligonucleotides for motor neuron diseases (ALS, SMA, SMARD1), neuropathies (CMT2A) and other neuromuscular disorders.
• Patient-specific 2D and 3D models: iPSC-derived brain and spinal cord organoids to study disease mechanisms and test new treatments.
• Neural stem cell transplantation and reprogramming of neuronal identity, in vitro and in vivo.
• Target discovery and functional analysis in a translational environment shared with clinicians experienced in clinical trials.

News & Events
July 2026 — ALS: the Policlinico di Milano licenses the miR-129 patent developed by our group to Life BioCeed, to bring a new therapeutic approach for amyotrophic lateral sclerosis towards clinical development
July 2026 — Our review on human 3D organoid models to gain mechanistic insight into motor neuron diseases is published in Nature Reviews Neuroscience (Frizzi et al.)
June 2026 — SOD1 pathogenic variants are more common than expected: our UK Biobank study of about 470,000 people, published in Annals of Neurology, identifies over 100 presymptomatic carriers and supports early intervention in ALS (Gagliardi et al.)
February 2026 — SMA begins during embryonic development: in Nature Communications, with Humanitas University and Columbia University, we show that early antisense oligonucleotide treatment rescues developmental alterations in SMA organoids (Faravelli et al.)
September 2025 — Rare SMN1 single-nucleotide variants identified in people with SMA (Neurology Genetics, Rimoldi et al.): genetic testing is key for diagnosis and access to therapies
October 2024 — Rare genetic diseases: Telethon Foundation funds three projects from University of Milan
July 2021 — SMARD1: a gene therapy clinical trial opened at Nationwide Children's Hospital in Columbus (USA)
Watch: Prof. Stefania Corti presents our new 3D model to fight ALS (Un Patrimonio per la Ricerca, Fondazione Patrimonio Ca' Granda).
