“Pioneering Efficient and Safe Advanced Therapies“
Our research group “Gene Therapy for Central Nervous System (GT4CNS)” is focused on the study of the molecular mechanisms involved in genetic diseases that cause nervous system dysfunction, with the final goal of developing effective and safe gene therapy strategies for those diseases.
By applying state-of-the-art techniques such as 2D and 3D cultures of iPSC-derived neurons from patients, in vivo gene therapy strategies in animal models (including transgenic models), -omic analysis (transcriptomic and spatial transcriptomic analysis), novel viral vectors, etc, our laboratory has three major complementary strategic research lines:
History. Our group was established in 2001 at the Biochemistry and Molecular Biology Department of the Universitat Autònoma de Barcelona (UAB) and is located on the 5th floor (H-Building) of the Institute of Neurosciences at UAB. Currently, it comprises a Research Lab and a Technological Platform (UPV) for Viral Vector Production, forming the UAB-VHIR Joint Unit. This Unit is affiliated with both the Universitat Autònoma de Barcelona and the Vall d’Hebron Research Institute (Barcelona).