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Gene therapy: a strategy to repair the injured CNS and PNS?

Although pharmacological and neurosurgical interventions have had some success, we believe that regeneration of the injured human spinal cord can be stimulated to a much greater extend by combining these approaches with gene and cell-based strategies.

The objective of this research line is to develop cell and gene-based repair strategies for the injured peripheral nerve, spinal roots and CNS.
The initial problems of cytotoxicity associated with adeno and herpes-based viral vectors have largely been solved during the last decade. Adeno-associated viral vectors (AAV) and lentiviral vectors (LV) direct long-term transgene expression in neurons and glial cells and this allows us to perform long term functional recovery studies to investigate the efficacy of cell and gene-based repair strategies. This project is executed in collaboration with Dr. Martijn Malessy, Neurosurgeon and Dr. Rob Hoeben, both at the Leiden University Medical Center.
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