Results for Preclinical Studies

Dr. Barzilay aims to understand what drives variability in the development of brain and behavior of youths growing up under stress. He studies biological and environmental mechanisms leading to suicidal behavior, using big datasets of diverse genotyped youths with deep phenotyping of environment.

Dr. Stachelek's research interests are in the cellular and molecular mechanisms that control vasculature inflammation.

Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.

Dr. Tasian's research centers on the bench-to-bedside investigation of precision medicine therapies for high-risk childhood leukemias.

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.

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. Kurre's laboratory has longstanding expertise in Fanconi Anemia (FA), a genetic condition with prominent hematologic complications. With training in transplantation and hematopoietic stem cell biology, he hopes to improve the understanding of the progressive hematopoietic failure in patients with bone marrow failure and FA, broaden diagnostic approaches, and develop next generation therapies.

Dr. Bhatnagar's research aims to further the understanding of the neural basis of individual differences in response to stressful experiences. This includes identifying neural substrates that produce resiliency or vulnerability to the effects of stress and determining treatments to mitigate vulnerability and to promote resiliency through both preclinical and translational studies.

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.