STUDY OF THE ROLE OF SMN IN MUSCLE RESIDENT PROGENITORS FOR THE IDENTIFICATION OF NOVEL THERAPEUTIC TARGETS FOR SMA
Principal investigator(s) :Dr. Martine Barkats
Institution :Institut de Myologie, Paris, France
Start year :2015
Duration :2 years
Muscle defects seen in SMA are partly due to the denervation and loss of the motor neurons that allow them to contract and relax. We know that these motor neurons die because they lack sufficient amounts of a protein called SMN. We also know that muscles react to this low level of SMN.
WHAT ARE THE RESEARCHERS AIMING TO DO?Q Does the muscle contribute directly to its own problems, independently of the motor neurons?
HOW WILL THE RESEARCHERS DO THIS?A Dr. Martine Barkats and her team will look at answering this question by:
- Looking at the role of SMN in the muscle’s “repair” cells (scientifically known as Satellite cells), which are stem cells that are triggered into action to repair any damage done to the muscle.
- If SMN is absent, do these repair cells still work?