Overview
Deciphering the Molecular Landscape of Neuromuscular Development in Spinal Muscular Atrophy
- Principal investigator(s):
- Dr. Morgan Gazzola
- Institution:
- I-Stem - Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, France
- Grant:
- €150,000
- Grant Type:
- Operating Grant
- Start Year:
- 2024
- Duration:
- 2 Years
- Call number:
- 12
- Status:
- Ongoing
Dr. Morgan Gazzola
Dr. Morgan Gazzola is a researcher at I-Stem (Institute for Stem Cell Therapy and Exploration of Monogenic Diseases) in France. He holds a PhD in Experimental Medicine from the Université Laval (Québec, Canada), where he studied airway hyperresponsiveness in asthmatic patients. After a first postdoctoral experience studying cytoskeletal dynamics in living cells, he has been working in the field of neuromuscular diseases for the last three years. His work focuses on the development of innovative three-dimensional cellular models to study the impact of neuromuscular disorders on the development of the neuromuscular system.
In Focus
Background
Although new treatments for SMA have been approved, many challenges remain in terms of cost, side effects and timing of use. To develop new combinatorial therapies, further efforts are needed to better understand the pathophysiology of SMA and identify new molecular mechanisms driving this pathology. Dr. Gazzola’s project will use advanced cellular and molecular techniques to investigate how the loss of SMN affects the development of the neuromuscular junction, the region where nerves and muscles connect.
How will Dr. Gazzola's team do this?
By differentiating human stem cells into complex mini neuromuscular organs (called neuromuscular organoids), the team aims to enhance understanding of SMA at the neuromuscular junction and explore changes in gene expression and DNA structure.
Why is it interesting to patients?
Ultimately, this project could identify novel molecular targets for the development of combined therapies, and improve outcomes for SMA patients.