Characterization of a Regulated Recombinant Adeno-Associated Viral Vector Encoding Human GDNF for the Treatment of Parkinsons Disease
Mandel, Ronald J., Ph.D.
Progress Report (as of 8/2002)
Numerous groups have now used recombinant adeno-associated viruses (rAAV) to add glial-derived neurotrophic factor (GDNF) to relieve the disorder in the brains of animals made parkinsonian, usually by MPTP injections. The problem before taking this therapy to human trials is that control of the amount of GDNF and the distances to which it is transmitted has yet to be determined. Dr. Ronald J. Mandel and his group at the University of South Florida used the antibiotic tetracycline to regulate transgene expression in parkinsonian rats, the element used consisting of a transactivator (the starter) and a silencer (the stopper).