Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production.
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.
Mandel
Ronald J
RJ
Department of Neuroscience, University of Florida College of Medicine, Gainesville, FL 32610, USA. rmandel@ufl.edu
Burger
Corinna
C
Snyder
Richard O
RO
D000818:Animals; D004345:Drug Industry; D015316:Genetic Therapy / Q000009:adverse effects; D005822:Genetic Vectors / Q000009:adverse effects; D006801:Humans; D010300:Parkinson Disease / Q000235:genetics / Q000628:therapy*; D010957:Plasmids / Q000235:genetics; D014780:Viruses / Q000235:genetics*