Gene therapy mediated seizure suppression in Genetic Generalised Epilepsy: Neuropeptide Y overexpression in a rat model
Kim L Powell, Xavier Fitzgerald, Claire Shallue, Valentina Jovanovska, Matthias Klugmann, Georg von Jonquieres, Terence J O'Brien, Margaret J Morris
NEUROBIOLOGY OF DISEASE | ACADEMIC PRESS INC ELSEVIER SCIENCE | Published : 2018
Neuropeptide Y (NPY) is an important 36 amino acid peptide that is abundantly expressed in the mammalian CNS and is known to be an endogenous modulator of seizure activity, including in rat models of Genetic Generalised Epilepsy (GGE) with absence seizures. Studies have shown that viral-mediated "gene therapy" with overexpression of NPY in the hippocampus can suppress seizures in acquired epilepsy animal models. This study investigated whether NPY gene delivery to the thalamus or somatosensory cortex, using recombinant adeno-associated viral vector (rAAV), could produce sustained seizure suppression in the GAERS model of GGE with absence seizures. Three cohorts of GAERS were injected bilater..View full abstract
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Awarded by NHMRC
This study was funded in part by NHMRC Project Grants to Margaret Morris (#568729), Terence O'Brien (#1059860) and Kim Powell (#1082215), and grant support from the Royal Melbourne Hospital Neuroscience Foundation.