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Report Summary

A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses


Study Summary

Overall Study Design 
Preclinical and clinical data stress the importance of pharmacologically-controlling glial cell line-derived 
neurotrophic factor GDNF intracerebral administration to treat PD The main challenge is finding a 
combination of a genetic switch and a drug which when administered at a clinically-approved dose reaches the 
brain in sufficient amounts to induce a therapeutic effect We describe a highly-sensitive doxycycline-inducible 
adeno-associated virus AAV vector This vector allowed for the first time a longitudinal analysis of inducible 
transgene expression in the brain using bioluminescence imaging To evaluate the dose range of GDNF 
biological activity the inducible AAV vector 80 x 10e9 viral genomes was injected in the rat striatum at four 
delivery sites and increasing doxycycline doses administered orally 

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    Lorraine Matheson
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    317-274-4519
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