Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows.
“The problem with too much clotting is by far one of the most staggering medical issues in the Western world,” Dr. Krishnaswamy said. See what he and his colleagues are doing to combat this deadly disease.
For children with hemophilia, every new research advance is a step toward a life filled with more activity, freedom, and adventure. The genetic condition, which affects roughly one in 5,000 births, causes children to bleed and bruise more easily than others ‚Äì meaning that a simple cut, scrape, or small surgery can result in uncontrollable and excessive bleeding. While hemophilia
Welcome back to another weekly edition of our roundup of research news from The Children's Hospital of Philadelphia! One of the things that is so exciting about research at CHOP is that our researchers are working to improve the health and lives of children in such a broad range of ways.
Research at The Children’s Hospital of Philadelphia never stops. To help keep you informed about new discoveries and CHOP researchers’ views on timely topics, we are bringing together a roundup of news highlights. Look for this as a recurring feature here on Cornerstone.
The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research in the Arruda Lab. Working collaboratively, the lab team and their colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.
The Camire Lab is interested in understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. The lab is also interested in developing novel therapeutic approaches (protein, gene-based, small molecule) to mitigate these events which are major causes of morbidity and mortality worldwide.
The research in the Sabatino Laboratory is focused on the inherited bleeding disorder, hemophilia. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.
The Gadue Laboratory studies human pancreatic and hematopoietic development and associated diseases using human pluripotent stem cells, including embryonic stem cells and induced pluripotent stem cells. The lab has devoted much of its research efforts on directed differentiation and CRISPR-based genome engineering of stem cells and is using this system for the study and development of treatments for diabetes and blood disorders.