Amyotrophic Lateral Sclerosis (ALS) is a severe neurodegenerative disease that still has no cure, progressively affecting the nerve cells responsible for movement. People living with ALS experience a gradual loss of vital functions such as walking, speaking, swallowing, and breathing. Despite advances in research, many questions remain about how the disease begins and the mechanisms driving its progression.
To address this challenge, researchers at the “Centro Dino Ferrari” are developing an innovative approach: creating three-dimensional laboratory models, called organoids, of the brain, spinal cord, and muscle, derived from cells donated by ALS patients. By connecting these “mini-organs,” it will be possible to reconstruct in vitro communication between the nervous system and muscles, opening new avenues to observe and understand disease mechanisms in a controlled environment.
A multidisciplinary approach combines 3D cell biology and spatial genomic mapping:
Generation and assembly of brain, spinal cord, and muscle organoids from ALS patient-derived cells cultured in 3D.
Measurement of electrical activity and muscle contractions to validate the functionality of the organoid systems.
Application of MERFISH to create detailed molecular maps of gene expression at single-cell resolution.
Dynamic monitoring of organoid models at 3, 6, and 9 months to study disease progression over time.
Screening of potential therapeutic molecules on functional organoids by tracking the molecular changes identified.
Early identification of molecular signals that precede symptoms, improving the potential for early diagnosis.
Development of targeted treatments that act on the initial causes of the disease, rather than just the symptoms.
Testing drug efficacy in a realistic human experimental system prior to clinical trials.
Providing an experimental platform that can also be applied to other neurodegenerative diseases.
Budget: €70,000
Prof.ssa Stefania Corti
E-mail: stefania.corti@unimi.it