Enhancers, one type of non-coding DNA regulatory element facilitating proper spatiotemporal gene expression, are fundamental to our understanding of development and disease. Majority of genetic variants, including single-nucleotide variants (SNVs) and copy number variants (CNVs) associated with disease traits, reside in/cover enhancers. It is imperative to improve our basic understanding of enhancer mediated gene regulatory logic so that we are better equipped to develop efficient gene therapies for genetic diseases.
The long-term goal of Yao’s research is developing cell type specific gene therapy for neurological disorders based on elucidating enhancer mediated regulation in neurodevelopment under physiological and pathological conditions, using cellular and mouse models. Specifically, Yao’s research focus on (1) Elucidating the function and behavior of enhancers, especially within the three-dimensional context of the nucleus, in central nervous system establishment; (2) Identifying and characterizing enhancer activity during neurogenesis: profile, characterize and modify enhancer activity and behavior during cell fate specification, using embryonic stem (ES) cells and induced pluripotent stem cells (iPSCs) derived cellular disease models; (3) Developing cell/tissue type specific gene therapy tools (genetically engineered exosomes packed with enhancer-driven CRISPR-Cas system) for genetic neurological diseases.
- Enhancer-mediated gene regulation
- Gene therapy
- Neurological disorders
- Pluripotent Stem Cells
- Genetic engineering via CRISPR-Cas system
- Publications by Yao Yao may be found at Pub Med.