BRAINVECTORS - from Brain Gene Transfer towards Gene Therapy: Pharmacological Assessment of AAV, CAV-2 and LVV

BrainVectors aims devising new gene therapy (GT)-based treatments for Parkinson's disease (PD), by delivering GDNF into the CNS with vectors derived from adeno-associated (AAV), canine adenoviruses (CAV-2) and lentiviruses (LV) with inducible gene expression. Although AAV, CAV-2 and LVY vectors have acceptable profiles in terms of biosafety, their immune response m ust be better characterized to develop these vectors for clinical use. The possibility to regulate expression represents a significant pharmacological progress of the gene therapy approach for PD. Brain Vectors will:
- identifying inducible gene expression cassettes with increased sensitivity of transactivators and inducers, will reducing the dose of drugs necessary to obtain GDNF expression;
- characterize the immune responses induced by the components of GDNF-AAV, -CAV-2 and - LVY in rodent models for PD by using biomarker-based immunological screening.

The project is based upon a network of 1 1 academic, private non-profit organizations and SMEs. The partners are linked in developing vectors backbones, vector production technologies and PD animal models. Others have strong immunological background, pioneering the biomarkers-based immuno-technologies for GT vectors, and have R&D expertise/facilities in/for animal cell technologies cGMP for biopharmaceuticals. 142 PM wi ll be involved during 48 months in R&D and TOK activities with 70 PM of recruited researchers and of 72 PM of seconded staff.