FAN Lab

Research

Epigenetic Studies of DNA Methylation in Development and Disease

Our laboratory investigates how epigenetic mechanisms, particularly DNA cytosine methylation, regulate neural development, brain function, and disease pathogenesis. We study how DNA methyltransferases (DNMTs), methyl-CpG binding proteins, and histone-modifying enzymes coordinate gene expression programs that govern neural stem cell differentiation, neuronal maturation, and neural plasticity. Using Cre/loxP conditional knockout mouse models and CRISPR-Cas genome editing, we generate transgenic models to dissect the functional consequences of altered DNA methylation in the central nervous system. Our work has revealed that DNA hypomethylation in neural precursor cells disrupts neuronal and astroglial differentiation and impairs postnatal neuronal survival. By elucidating epigenetic mechanisms underlying neurological and developmental disorders, we aim to identify novel therapeutic strategies targeting epigenetic regulation.

Stem Cell Technology: Neural Differentiation and Organoids

To bridge mechanistic studies with human disease, we employ advanced stem cell technologies, including induced pluripotent stem cells (iPSCs) and lineage-directed differentiation systems. We generate disease-specific human iPSC models to investigate how epigenetic dysregulation contributes to neurodevelopmental and neurogenetic disorders such as Fragile X syndrome, ICF syndrome, and Tatton-Brown-Rahman syndrome. Through controlled differentiation into neural lineages and three-dimensional brain organoid models, we examine cell-type–specific epigenetic regulation during development and disease progression. These platforms allow us to recapitulate key aspects of human brain development in vitro and provide powerful systems for mechanistic studies and therapeutic testing.

Development of mRNA Delivery Technologies for Rare Diseases

Building on our understanding of gene regulation and disease mechanisms, our laboratory is developing mRNA-based therapeutic strategies for rare genetic disorders. We focus on designing and optimizing mRNA delivery systems that enable efficient, targeted, and controlled expression of therapeutic proteins in relevant tissues. By integrating molecular engineering, delivery platform optimization, and disease modeling, we aim to translate mechanistic insights into innovative treatment approaches. These efforts represent a critical step toward precision medicine for rare and currently untreatable diseases.