Overview
Skeletal Muscle Stem Cells as untapped therapeutic targets for SMA long-term treatment (SATSMA)
- Principal investigator(s):
- Dr. Nathalie Didier
- Institution:
- INSERM, France
- Grant:
- €120,000
- Grant Type:
- Operating Grant
- Start Year:
- 2024
- Duration:
- 2 Years
- Call number:
- 12
- Status:
- Ongoing
Dr. Nathalie Didier
Dr. Didier is a researcher at the French Blood Establishment, working in an INSERM (Institut national de la santé et de la recherche médicale) laboratory, in France. She holds a PhD in Cellular and Molecular biology (University Paris XI). She has a long-standing interest in understanding the molecular mechanisms governing muscle stem cells under physiological and pathological conditions. She was awarded a grant from SMA Europe to dissect the role of these cells in SMA physiopathology and their fate under risdiplam treatment.
In Focus
Background
While the approved disease-modifying therapies for SMA have provided significant benefits for people living with this condition, the long-term effects of these treatments remain unknown, and results are more variable for late-treated patients and those with milder forms. Therefore, it is crucial to better define SMN protein requirements and the effects of the treatments in all tissues, especially muscle, which is particularly affected by the disease. Dr. Didier and her team discovered that muscle stem cells (MuSCs) have a high requirement for SMN, and that a lack of SMN in these cells is sufficient to induce motor neuron death. Given the key role of MuSC in maintaining muscle tissue and its ability to repair itself, it is essential to determine whether the approved therapies are able to preserve these cells.
How will Dr. Didier’s team do this?
Dr. Didier, in collaboration with Dr. Piera Smeriglio, will focus on better understanding the link between MuSC and motor neurons, and exploring whether and how oral risdiplam treatment acts on these cells.
Why is it interesting to patients?
With this project, the future goal is to develop combinatorial therapeutic strategies to ensure the integrity of the whole neuromuscular system throughout the life of treated patients.