Funded Research Projects


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
Institut de Myologie, Paris, France
Start Year:
2 years
SMA Europe

Dr. Martine Barkats

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.

In Focus

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.