Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.
Dr. Ackermann studies diabetes (types 1 and 2) and congenital hyperinsulinism using mouse models, cell lines, and primary human tissue. She aims to identify novel pathways regulating beta cell insulin secretion, leading to innovative therapeutic strategies for these disorders. Current studies include in vivo mouse physiology, ex vivo human islet physiology, CRISPR-Cas9 gene editing, epigenetic modification, and single-cell functional genomics.
Dr. Shah's research is centered on understanding obesity and its related complications. Her current work includes clinical and translational studies exploring pathophysiology and modulation of obesity-related adipose tissue and systemic inflammation using human cell lines and clinical trials. She is also involved in clinical studies of outcomes and risk factors of polycystic ovarian syndrome and type 2 diabetes in teens.
Dr. Foster’s current research focuses on immunotherapy for pediatric solid and brain tumors. Specifically she is investigating chimeric antigen receptor (CAR) T cell therapy for neuroblastoma, high-grade gliomas, medulloblastomas, diffuse intrinsic pontine gliomas, and other brain tumors. The goals of her research are to develop pre-clinical CAR T cells for translation into clinical trials to help these devastating tumors.
Dr. De Raedt researches pediatric high grade glioma development and aims to understand the involvement of crucial pathways. He investigates pathway interaction, and explores ways to develop therapies through analyzing human tumors, performing cellular studies, and developing accurate mouse models. This allows Dr. De Raedt and his team to perform novel pre-clinical studies that can lead to clinical trials.
The Raymond G. Perelman Center for Cellular and Molecular Therapeutics has established state-of-the-art Current Good Manufacturing Practices (cGMP) clinical vector manufacturing suites for adeno-associated virus (AAV) and lentiviral (LV) vectors.
The Wolfson Family Laboratory for Clinical and Biomedical Optics focuses on the development and use of novel non-invasive optical devices to probe cerebrovascular hemodynamics and physiology in vivo. These devices are used in clinical and pre-clinical studies to discover the timing and causes of brain injury during care.
The Cancer Pharmacology Laboratory supports clinical trials of new mechanistically targeted and immune-based anticancer drugs by conducting pre-clinical and clinical pharmacology studies of the agents. The lab also studies conventional anticancer drugs to develop more rational and effective dosing methods, and the lab team is developing circulating biomarkers of organ function, drug toxicity, and tumor burden as clinical trial endpoints.
The Mitochondrial Medicine Research Laboratory investigates the genetic etiologies, pathophysiologic mechanisms, and therapeutic targets of mitochondrial disease. The lab applies state-of-the-art technologies and approaches in diverse animals (C. elegans worms, D. rerio zebrafish, M. musculus mice), human cells, and patients to cross-validate key translational insights that will enable precision diagnosis, monitoring, and therapeutic management of primary and secondary mitochondrial diseases.