Over the last 15 years, the McCown laboratory has focused upon the development of safe, effective gene therapies for the treatment of epilepsy. Over these years we have developed a novel gene therapy platform where inhibitory neuroactive peptides could be expressed and constitutively secreted from AAV transduced neurons. This novel platform has proven capable of blocking both electrographic and behavioral limbic seizures. In addition, we have developed a means to create novel AAV vectors that upon peripheral administration, selectively cross the seizure compromised blood brain barrier but do not cross the intact blood-brain barrier. The ability of these hybrid adeno-associated virus vectors to selectively target areas of brain pathology could radically shift the application of viral vector gene therapy, not only for the epilepsies but also other neurological disorders that compromise the blood brain barrier, such as Parkinson’s disease.
Associated Grants
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The Development of Novel Adeno-associated Virus Vectors for Intravenous Parkinson's Gene Therapy
2011