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 lab focuses on translational target discovery for a range of neurodegenerative diseases. We combine technology development of large-scale CRISPR-based perturbation screens with application of such technology together with additional genomic approaches.
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.
Our research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule or gene therapy based strategies for therapy. In recent work, we demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. We continue to develop novel vector systems to improve therapeutic outcomes.