Projects

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. 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
    Grant :
    €124,000
    Start year :
    2015
    Duration :
    2 years
    Status :
    Ongoing


    Dr Martine Barkats - square - smMuscle 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?
    The long term aim is to identify new targets specifically affected in SMA satellite cells, and see if gene therapy targeted at these cells would benefit the muscle damage seen in SMA.

    ABOUT DR. BARKATS

    Dr. Martine Barkats heads up her laboratory at the Institut de Myologie in Paris. To find out more about her group and her work, please follow the link here.