Adenovirus for neurodegenerative diseases: in vivo strategies and ex vivo gene therapy using human neural progenitors.

The discovery of major neurodegenerative mechanisms has opened the way to the development of novel therapeutic approaches. Gene therapy now enables researchers to overcome certain problems inherent to pharmacotherapy and to the grafting of embryonic cells. The production of recombinant adenoviruses are promising for in vivo gene therapy involving neuroprotective (Ad-SOD), neurotrophic (Ad-NGF) as well as restorative (Ad-TH) strategies. In addition, human neural progenitors offer great potential as vehicles for ex vivo gene therapy to replace degenerated cells in advanced stages of neurodegenerative diseases. This paper describes the clinical values of the new generations of adenoviral vectors.