A New Era of Research
Our research teams solve the most challenging problems in children’s health. How do we make these breakthroughs? Our scientific community thrives on collaboration from bench to bedside that leads to effective therapies, medical devices, and treatment techniques. When we have an important pediatric research question to answer, our lab investigators produce novel understanding about basic life processes, and our physician-scientists use these insights to inform patients’ clinical experiences. We align that expertise with our first-of-its-kind multidimensional data collection. The result is unparalleled knowledge creation. From laboratory discoveries like personalized cellular immunotherapy treating childhood cancer, to the development of new vaccines preventing disease globally, to establishing public polices improving children’s safety, our work is transforming our patients’ and their families’ lives.
Our Major Scientific Themes
We ask novel research questions that bridge disciplines in targeted scientific areas and span the research continuum to accelerate scientific discoveries that improve the health of children.
The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research by Dr. Arruda. Working collaboratively, Dr. Arruda and his colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.