Danwei Huangfu, a research fellow in the Sloan Kettering Institute’s Developmental Biology Program, describes genome sequencing and its use in regenerative medicine. She explains how her lab is using human pluripotent stem cells (hPSCs) to model the process of disease progression, which is crucial to the development of regenerative treatments. Understanding the genetics of the patient is critical and one way to figure out the workings of the disease is sequencing the tumor’s genome.
Human genome sequencing technology has evolved rapidly over the past few years and Dr. Huangfu says that her lab is seeking to make these technologies more cost effective so that more studies to be conducted. One of the purposes of sequencing is to identify mutations in the genome and find out if they are directly related to diseases such as diabetes and cancer. Her team is analyzing hPSCs to model human disease phenotypes. These cells have the ability to differentiate to all cells in the human body, giving researchers a powerful tool to work with. Human pluripotent stem calls can also be regenerated in a laboratory setting.
She talks about ways hPCSs can be made in the lab, such as defined factor reprogramming and nuclear reprogramming. One of the eventual goals for these tools is to coax manufactured cells and into developing into the specific cells that a patient is missing.
Dr. Huangfu discusses current technology in disease modeling and her work in editing the genome to better study specific mutations. This type of precise genome editing has been very difficult in the past, but there have been recent technological breakthroughs. Using diabetes as an example, she shows the steps researchers take to use this technology to study the disease and the ways they can use hPSCs to help find cures. She also talks briefly about efforts to replicate pancreatic tissue and to introduce specific mutations into the genome that would allow researchers to study the cellular genesis of pancreatic cancer in a much more controlled way.