A clinical-grade gene therapy vector for pharmacoresistant epilepsy successfully overexpresses NPY in a human neuronal cell line.
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Abstract | :
Epilepsy is a common neurological condition characterised by recurrent unprovoked seizures and often treatable with appropriate medication. However, almost 30% of cases are pharmacoresistant and while a proportion of these may be amenable to resective surgery, a gene therapy approach could be an attractive alternative option. Neuropeptide Y (NPY) has anticonvulsant and anti-epileptogenic properties in animal models of temporal lobe epilepsy when delivered by an adeno-associated viral (AAV) vector. Here we sought to demonstrate successful secretion of NPY from AAV-transduced human neuronal cells, which would be essential in planning any clinical trial. |
Year of Publication | :
2017
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Journal | :
Seizure
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Volume | :
55
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Number of Pages | :
25-29
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Date Published | :
2017
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ISSN Number | :
1059-1311
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URL | :
http://linkinghub.elsevier.com/retrieve/pii/S1059-1311(17)30350-3
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DOI | :
10.1016/j.seizure.2017.12.005
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Short Title | :
Seizure
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