iBB

Cerebellar ataxias are characterized by progressive neurodegeneration of the cerebellum. Current knowledge about the disease is based on postmortem studies and animal models that do not fully recapitulate the features of human ataxias. The development of disease models is thus an important unmet medical need. The goal of the project "CEREBEX", which has recently been approved for funding by PORLisboa, is to generate 3D cultures of mature cerebellar neurons to investigate ataxia mechanisms and therapeutic strategies. The project, which falls within the scientific area of Medical Biotechnology, is headed by Joaquim Cabral from SCERG-iBB and involves the collaboration with the Institute for Molecular Medicine. The proposal "CEREBEX - Generation of gene-edited cerebellar organoids for ataxia research" was submitted in the context of the 2017 Call for SR&TD Project Grants.