Gene therapy is emerging as a new treatment modality for many human diseases, by transferring DNA to an individual to restore and manipulate a defective gene to mitigate disease. The treatment strategies are divided into two main categories i) in vivo gene delivery using a modified viral vector and ii) ex vivo engineering cells for autologous transplantation.

Over the last few years, CRISPR/Cas technology has revolutionized biomedical research by creating genetically modified cell and animal models; and more importantly, it has extended our ability to target complex human diseases with a safer genomic intervention in specific cells or tissues. The Feng Bo lab is dedicated to improving and expanding the CRISPR/Cas technologies for in vivo and ex vivo gene therapies through fundamental discoveries and innovative applications. There is a particular emphasis on genetic disorders, such as haemophilia B and β-thalassemia, and engineering immune cells for cancer therapy. 

We believe comprehending the underlying mechanisms of gene targeting in various biological contexts (stem cells vs. somatic tissues, in vivo vs. in vitro) using advanced molecular and informatics approaches will lead to important discoveries that will facilitate us to develop novel therapeutic strategies to treat currently incurable human diseases.