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A clinical-grade gene therapy vector for pharmacoresistant epilepsy successfully overexpresses NPY in a human neuronal cell line.

Author
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
Journal
:
Seizure
Volume
:
55
Number of Pages
:
25-29
Date Published
:
2017
ISSN Number
:
1059-1311
URL
:
http://linkinghub.elsevier.com/retrieve/pii/S1059-1311(17)30350-3
DOI
:
10.1016/j.seizure.2017.12.005
Short Title
:
Seizure
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