Journal
EXPERIMENTAL NEUROLOGY
Volume 209, Issue 1, Pages 58-71Publisher
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.expneurol.2007.08.008
Keywords
adenovirus; adeno-associated virus; herpes simplex virus; lentivirus; purification; quality control; quality assurance; cleanroorn
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Funding
- NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE [P01NS036302] Funding Source: NIH RePORTER
- NINDS NIH HHS [P01 NS363602, P01 NS036302-06A10004, P01 NS036302] Funding Source: Medline
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Because Parkinson's disease is a progressive degenerative disorder that is mainly confined to the basal ganglia, gene transfer to deliver therapeutic molecules is an attractive treatment avenue. The present review focuses on direct in vivo gene transfer vectors that have been developed to a degree that they have been successfully used in animal model of Parkinson's disease. Accordingly, the properties of recombinant adenovirus, recombinant adeno-associated virus, herpes simplex virus, and lentivirus are described and contrasted. In order for viral vectors to be developed into clinical grade reagents, they must be manufactured and tested to precise regulatory standards. Indeed, clinical lots of viral vectors can be produced in compliance with current Good Manufacturing Practices (cGMPs) regulations using industry accepted manufacturing methodologies, manufacturing controls, and quality systems. The viral vector properties themselves combined with physiological product formulations facilitate long-term storage and direct in vivo administration. (c) 2007 Elsevier Inc. All rights reserved.
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