Targeting multi-organ defects for the treatment of spinal muscular atrophy
Spinal muscular atrophy (SMA) is a motor neuron disease caused by reduced levels of the survival of motor neuron (SMN) protein. The SMN protein is ubiquitously expressed in the central nervous system (CNS) and peripheral organs. Restoring SMN protein levels in the CNS constitutes a potent strategy that has led to the approval of therapies for clinical use. The aim of the EU-funded SMABEYOND project is to investigate the role of the SMN protein in peripheral organs and determine the intrinsic defects encountered in SMA patients. The ultimate goal is to develop SMA patient-derived disease models and test the therapeutic effect on peripheral organs of systemic SMA therapy.
Call for proposal
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Funding SchemeMSCA-ITN-ETN - European Training Networks
WC1E 6BT London