Developmental and Cell Biology

Developmental biology is the study of inductive initiation and the construction of tissues, organs, and ultimately, the formation of the intact individual. Between fertilization and adulthood, a myriad of changes under genetic control occur which result in the development of a unique individual from a single fertilized cell.

For the translational pediatric investigator, abnormalities of development – disorders that affect tissue differentiation, organogenesis and morphogenesis – constitute a significant component of human hereditary disease. Indeed, developmental biology is the quintessential basic science of pediatrics, as 60 percent of chronic disease in children are from abnormalities in development. Although not all critical development pathways have been delineated, there appear to be a finite number of genetically determined programs that fail to operate correctly in the production of common developmental disorders.

Cell biology is an enabling technology as well as a research theme. Each organ of the body is comprised of multiple cell types, where congenital and acquired diseases are often found. The comparative and detailed study of the precise cells adversely affected by disease permits delineation of critically affected intracellular signaling pathways. Such data can lead to the identification of key molecules that when modified or replaced can provide targeted therapies for the translational researcher. Cell biological approaches provide a functional read-out for genomics and can be applied at several levels of organization. Examples include the identification of the functional effects of a single normal or defective gene product, the determination of how different protein products interact with one another in complexes and networks, or the definition of the influences of different cell types upon one another within an organ during specific stages of normal and abnormal development.

Examples of developmental and cell biology research underway at Children's Research Institute include:

• One of two national Research Centers of Excellence in Pediatric Nephrology.
• Identification of new molecular targets and technologies for treating defects of kidney development.
• Innovative studies using the zebrafish model to study the action of genes on organ development.
• A study of early neurocrest cells and their potential role in childhood malignancy or malformation.
• Development of histology and imaging cores to support the developmental and cell biology investigators.

Developmental cell biology approaches will enable the fundamental analysis of the mechanisms causing disease in children. The in-depth examination of novel cellular pathways will lead to the identification of specific key molecules to be targeted for new therapies by innovative delivery technologies. This will ultimately facilitate early intervention and prevention of disease in children.