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. The ROCK-ERK signalling network in SMA – mediator of neurodegeneration and SMN independent treatment target

    Principal investigator(s) :
    Dr. Niko Hensel
    Institution :
    Institute of Neuroanatomy, Hannover Medical School, Germany.
    Grant :
    Grant Type:
    Call number :
    Start year :
    Duration :
    2 years
    Status :

    ROCK-ERK signalling pathway in SMA

    Dr. Niko Hensel, Institute of Neuroanatomy at Hannover Medical School in Germany, has been granted an SMA Europe award to investigate the ROCK-ERK signalling network in SMA. Spinal Muscular Atrophy is a disease characterised by a loss of muscle control. This is caused by degeneration of specific cells in the spinal cord, the motor neurons. Cell death and survival is controlled by two proteins called ROCK and ERK. In SMA, they exhibit an enhanced activity.

    Dr. Hensel will look at the effects of reducing  their activity back to normal levels in SMA model mice which have similar symptoms to human patients. Dr. Hensel will use two different approaches. First, he will use drugs which are known to inhibit ROCK and ERK and which have already been approved to treat other diseases. In addition, he will use antisense oligonucleotides enabling him to more specifically down regulate activities of the ROCK and ERK proteins.