BRAINVECTORS · From Brain Gene Transfer Towards Gene Therapy: Pharmacological Assessment of AAV, CAV and LVV""

BRAINVECTORS aims devising new gene therapy(GT)-based treatments for Parkinson’s and other neurodegenerative diseases, in substitution of current systemic treatments, by delivering neurotrophic factors (GDNF) into the CNS with new vectors derived from adeno-associated (AAV), canine adenoviruses (CAV) and lentiviruses (LV) with inducible gene expression.Although AAV, CAV and LVV are considered acceptable in terms of bio-safety, their immune response must be well characterized in order to further develop these vectors for clinical trials. Furthermore, the possibility to switch-off the expression of neurotrophic factors in case of adverse effects represents a significant pharmacological progress of the gene therapy approach for Parkinson’s disease. BRAINVECTORS will:- devise new inducible gene expression cassettes with increased sensitivity of transactivators and inducers reducing thus the dose of drugs necessary to obtain GDNF expression in brain