Our lab investigates the dual role of extracellular vesicles (EVs) in neurodegenerative diseases—both as mediators of pathogenesis and as potential therapeutic agents. EVs are nanosized, membrane-bound vesicles released by virtually all cell types, facilitating intercellular communication by transporting molecular cargos. We utilize cellular and animal models to dissect disease mechanisms, integrating advanced molecular biotechnology, genetics, and epigenetics with state-of-the-art genome editing tools. Our goal is to uncover novel insights into disease pathology and develop innovative EV-based therapies for human and animal health.
Enhancer Mediated Transcription Regulation
Enhancers, one type of non-coding DNA regulatory elements facilitating proper spatiotemporal gene expression, are fundamental to our understanding of development and disease. Majority of genetic variants associated with disease or agronomic traits, reside in/cover enhancers. We are using high throughput genomic tools combing with mouse genetics to identify and elucidate enhancer mediated gene regulation during muscle progenitor cell differentiation and iPSC-derived neural cell types. The outcomes will equip us to develop efficient gene therapies for genetic diseases and strategies for agronomic improvements.
Delivery of Genome Editing
Delivery of genome editing tools in vivo in a safe and efficient way is perhaps one of the biggest barrier to fulfill its potential in biomedical and agriculture fields. We are developing novel delivery shuttle, such as genetically engineered exosome, for CRISPR mediated genome editing to facilitate its application in gene therapy for neurodegenerative diseases and muscle disorders.
Gene Therapy
With the accumulated knowledge of genetic basis of diseases and recent innovations in genome editing tools, especially the CRISPR-Cas9 system, gene therapy is becoming an attractive treatment for devastating genetic diseases, including neuromuscular diseases (ALS). We are establishing iPSC and mouse model of ALS and other muscle disorders to screen for therapeutic targets and validate CRISPR mediated gene therapies. (genengnews.com)
Interested? Join Us!
The Yao lab is actively seeking graduate/undergraduate students, technician and postdoctoral fellows. Contact us if you are interested in joining.
Interested? Join Us!