Results for Gene Therapy

Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.

Dr. Freedman has a particular interest in epidemiologic and translational research within pediatric supportive oncology. He aims to advance the understanding and treatment of symptoms, infections, and organ toxicities in pediatric cancer and hematopoietic stem cell transplant patients.

Dr. Boris is interested in the clinical outcomes in patients with postural orthostatic tachycardia syndrome, an increasingly recognized disorder of autonomic nervous system dysfunction that is estimated to affect as many as one to three million American children and adults.

Dr. Gonzalez-Alegre's long-range research goal is to advance the application of precision medicine in the neurology clinic. His research focus revolves around genetic disorders that affect the brain, spanning from the diagnosis of novel genetic disease in the clinic to the identification of novel molecular targets using disease models and the design of early-phase human clinical trials.

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. Nah-Cederquist investigates solutions to clinical problems in pediatric plastic surgery. Her lab is built around the strengths of CHOP's clinical practices and basic science research. This offers the unique opportunity to directly test hypotheses born from clinical problems in the laboratory, and to take new technologies and concepts developed in the laboratory to patient care.

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

Dr. Anderson’s research interests focus on the molecular and cellular mechanisms that govern the development of the mammalian forebrain. In his research on the development of the cerebral cortex, he is particularly interested in understanding the molecular underpinnings behind the fate determination and axon targeting of subclasses of GABAergic interneurons implicated in the neuropathology of schizophrenia.

Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis.

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.