Research

Gene therapies offer powerful new options for addressing previously fatal or untreatable genetic diseases. Our investigators are at the forefront of pre-clinical research, vector development and clinical trials of gene therapies for a broad range of pediatric disorders.

Childhood cancers are driven by different genetic changes than adult cancers. Our scientists interrogate their complex origins with advanced genomic and drug screening techniques, and partner with clinical researchers to drive a new generation of pediatric cancer trials.

Understanding the nervous system at the genetic, molecular, cellular and systems level opens opportunities to tackle hard-to-treat disorders. From disease modeling in human neurons to clinical trials for genetic conditions, our basic and clinical scientists collaborate closely.

Next-generation DNA sequencing, chromosomal microarray, gene-expression studies, gene editing, genome-wide association studies and other tools are bringing fresh insights into the basic biology of disease, from common conditions like obesity to the exceedingly rare.

New tools are fueling a burst of discoveries in immune deficiency, allergy, autoimmunity and inflammatory disease. We are learning new immunization strategies and how the immune system influences diseases not classically seen as immunologic, from neurodegenerative disorders to heart disease.

Our labs are creating pluripotent stem cells for disease modeling and drug screening, investigating natural regenerative processes with cellular “barcodes,” and developing cell-based therapies and testing chemical factors that stimulate regeneration pathways.