
The GeN (Genetics and Neurobiology) laboratory investigates the molecular and genetic basis of brain disorders, with a particular focus on Alzheimer’s disease, neurodegeneration, aging, and rare forms of autism. Our research is driven by a strong interest in uncovering the genetic components of memory using transcriptome-wide data from single cells, linking gene expression and epigenetic information to neurodegenerative phenotypes. More broadly, GeN focuses on understanding the neurobiology of memory and the mechanistic basis of neurological disorders such as autism, rare forms of autism, neurodegeneration, and aging.
Our work combines fundamental biology with translational research, leveraging both conventional and unconventional animal models that naturally develop disease-relevant pathology to uncover conserved mechanisms of vulnerability and resilience. To address these questions, we employ an interdisciplinary approach that integrates molecular biology, genetics, evolutionary genetics, and viral tracing.
A central pillar of our translational platform is the FRAXA–Drug Validation Initiative (FRAXA-DVI), through which we systematically evaluate therapeutic candidates for rare Fragile X syndrome. This programme provides a robust preclinical framework that bridges discovery and clinical translation for rare forms of autism, including Kabuki syndrome, Pitt-Hopkins syndrome, and Angelman syndrome.
In parallel, GeN works closely with industry partners to develop and test novel compounds for rare and complex neurological diseases, including Alzheimer’s disease. By integrating artificial intelligence-driven approaches with experimental validation, we aim to accelerate therapeutic discovery and improve translational outcomes.
GeN operates at the interface of genetics, neuroscience, and drug development, with a clear objective: to transform fundamental biological insights into tangible advances for patients. Our research programme is supported by competitive grants and private funding, and is strengthened by an extensive network of industry partners and national and international collaborators.

Lab Members
Grants
Fondecyt Regular, FRAXA Research Foundation, Kabuki Research Foundation, National Institute of Health (NIH), USA and work with the pharmaceutical Industry
Collaborations


