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Erik Rossi

Sobre

PhD in Human and Experimental Pathology, working in Molecular Neuroscience. His project focused on investigating pathogenic variants of the SCN2A gene associated with Autism Spectrum Disorder, using hiPSC-derived neuronal models and multiomics approaches. His research integrates strategies for gene modulation, including CRISPRa and cis-regulatory editing, with an emphasis on developing innovative therapies. He is currently working on advancing gene-delivery platforms based on extracellular vesicles, aimed at functional restoration in human neurons.

Áreas de Pesquisa:

Cellular and Molecular Biology

About the research:

The project explores the use of engineered extracellular vesicles as an innovative delivery platform for gene modulation in the central nervous system. The proposal aims to develop technologies capable of carrying in vivo editing and transcriptional regulation tools, such as CRISPRa and base editors, with specific targeting of neuronal cells. The vesicles are designed to optimize molecular loading, cellular specificity, and brain biodistribution, increasing the efficiency and safety of interventions. By integrating molecular engineering, neurobiology, and preclinical validation, the project aims to establish a new generation of therapeutic strategies for neurological and neurodevelopmental diseases.

 

Awards and honors:

🏆 CCM Research Incentive Award, Congress on Brain, Behavior and Emotions
🏆 Best Academic Doctoral Thesis, Instituto Gonçalo Moniz

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