The neuroregeneration group led by joost Verhaagen was awarded with a ERA-NET grant of 225.000 euros for his research on spinal cord repair, entitled AxonRepair. The project is aimed on releasing the neuron-intrinsic brake on axon regeneration.
Spinal cord injury (SCI) leads to permanent disability and is a significant clinical problem with 130.000 new cases/year worldwide. Following SCI long axon tracts fail to regenerate. This is the primary reason for the sustained loss of bodily functions.
AxonRepair aims to develop a strategy to promote long-distance axon regeneration and functional recovery after SCI by reprogramming neurons into a regenerative state and by overcoming the axon transport block that is a barrier to axon regeneration. Joost Verhaagen: “In AxonRepair we bring together world-class experts on the transcriptional control of axon regeneration, axon transport and therapeutic gene delivery. We already have identified factors that promote CNS axon regeneration and modest sensorimotor recovery in animal models of SCI. We now seek to combine these factors to deliver greater axon regeneration and clinically significant functional recovery. We will use clinically translatable adeno-associated vector-based gene delivery, novel techniques to quantify axon trajectories in cleared spinal tissue and innovative, automated methods for assessing dexterity in rats”.
Work package (WP) 1 aims to reprogram neurons into a regenerative state by testing the effect of combinatorial delivery of key transcription factors identified in our collective’s discovery pipelines. WP 2 aims to restore axonal transport of growth-related receptors (integrins) to injured spinal axons. WP 3 aims to provide novel mechanistic insight into how interventions at the transcriptional and axon transport level work and whether these interventions act synergistically in rat models of SCI. At completion of the project Verhaagen expects to have developed a method to release the brake on axon regeneration. The participation of the world-leading gene therapy company uniQure ensures that newly generated know-how will be exploited rapidly by entering into a co-development agreement and/or intellectual property strategy.