Javier Ruiz y Fermín Moreno. Grupo de Enf. Neurodegenerativas - IIS Biodonostia.
Salón de Actos-IIS Biodonostia.
Frontotemporal dementia and Parkinson disease are neurodegenerative diseases that cause progressive functional impairment. Disease-modifying treatments are lacking. In this seminar we will present clinical trials offered in our center for FTD-GRN and PD.
In PD we will focus in an upcoming ASO-based clinical trial. In FTD-GRN we are participating in a clinical trial that uses AAV-9 as a vector delivery of the GRN gene to the CNS. We will review the advantages and challenges of rAAV-based therapies with focus in genetic frontotemporal dementia.
We will explain how this clinical trials try to resolve usual problems of these therapies, like appropriate delivery to the brain, immunogenicity and others. rAAV-based therapies will probably be the future of gene-therapy in neurodegenerative diseases, but they are already the present in our clinical trials.