Medicine has transformed the lives of individuals, families, and societies. It has shaped the world as we know it, and continues to do so in surprising ways. Many of these advances are the result of curiosity-driven research – the very human impulse to dive into the unknown to discover something new or understand “why.”
Research in HSCRB is firmly rooted in discovery biology: asking fundamental questions to understand the workings of life. The knowledge we build brings us ever closer to combating disease and tissue degeneration, and to improving human health.
Our work serves as a bridge between discovery and application, opening up new approaches to treating disease, and giving rise to new technologies that improve and accelerate biomedical research.
When you visit our faculty page, you will see the full range and depth of expertise in our research program. Here, we present just a few examples.
Nervous System Diseases
At some point in our lives, many of us will be affected by neurological disease. That stark reality motivates HSCRB researchers to investigate how the nervous system is made and the workings of neural networks that govern behavior. They build on this work to seek new avenues to combat diseases like Alzheimer’s, Parkinson’s, autism, and ALS from every direction.
Human Models of Disease
Using model organisms such as zebrafish, axolotl, and mouse, we seek to understand how tissues and organs form, grow, and repair. Today, we are also able to study human diseases in human tissues far more accurately than ever before.
HSCRB scientists were the first to create motor neurons in a dish using induced pluripotent stem (iPS) cells from patients. We continue to leverage human pluripotent cells as a powerful tool to understand disease and study the effects of drugs on a patient’s cells directly in the lab.
Our deep dive into the molecular biology of pancreatic organ development has resulted in our ability to make millions of insulin-producing cells in a dish. This is transformative for diabetes research, creating a direct bridge from the bench to the clinic.
Organoids are changing the way we study human diseases. For example, brain organoids allow us to explore human brain development directly, getting to the root of conditions like autism and schizophrenia. Only recently, HSCRB researchers developed a method to create much more reproducible brain organoids, which vastly improves the utility of these tools. They can now be used to investigate sophisticated aspects of human brain development and neurodevelopmental disease that would have been impossible only a year ago.
Immunology in Focus
HSCRB research intersects with immunology in a range of contexts, from bone marrow transplants to tissue repair to autoimmunity. In addition to studying the immune system’s complex roles in health and disease across different organ systems, we explore key aspects of reproductive immunology and cancer so that we can understand how biological material that should be treated as ‘foreign’ by the body is given a free pass by immune cells.
One goal of research at HSCRB is to apply this knowledge to engineer cells to protect them in the settings of allogeneic transplantation and autoimmunity. For example, our pioneering work to generating replacement beta cells is complemented by our research into how they can be protected from immune attack, and our investigation into the autoimmune response in diabetes patients.
Biologists, clinicians, technologists, physicists, computational scientists, software engineers, and mathematicians throughout the world are working together to map every type of human cell: where each type is located in the body, the genes it expresses, and what part it plays in complex biological networks. This work, spearheaded by projects like the Human Cell Atlas, will change the way we understand human health, and how we diagnose and treat disease.
Attaining such an ambitious goal depends on new approaches in single-cell genomics, which presents unique experimental and computational challenges. HSCRB scientists collaborate across sectors to create robust, reproducible methods that make it possible to profile individual cells far more quickly and precisely than ever before.
Generation and Regeneration
All stem cells are self-renewing but over time, they lose their ability to repair. For many vital organs, regenerating lost tissue is either an extremely slow process or doesn’t happen at all. HSCRB scientists are investigating how tissues and organs form, how they respond to insult and injury, how they repair, the mechanisms behind those processes, and how different drugs might influence them. This knowledge is fundamental to influencing the repair process in specific tissues.
By comparing young and old animals, across organ systems, HSCRB researchers have identified circulating factors that can restore function to damaged tissue. These findings have sparked a new generation of cross-sector research that has implications for human diseases associated with aging. HSCRB researchers are also investigating how salamanders heal their wounds without scarring, how they regenerate lost limbs throughout life, and what pathways they activate to call up the right cells and direct the repair. They also investigate pathways involved in stress response, which impact everything from hair color to wound repair.