In Vivo: New Modeling System for Disease Development

Whitehead Institute researchers have created a new mouse-human modeling platform that could be used to study neural crest development as well as the modeling of a variety of diseases, including such cancers as melanoma and neurofibromatosis.

“We introduced human committed stem cells at the right stage into the mouse embryo in utero and had them integrate into developing tissues,” says Whitehead Founding Member Rudolf Jaenisch. “The results are encouraging and provide a proof of principle — an important first step toward the goal of generating mice that carry disease-relevant human cells in the relevant tissue.”

Resulting mouse-human chimeras would fill an important gap in disease research, as existing models do not accurately mimic certain diseases and disease states. Cancer is frequently studied by implanting cells from human tumors into mice, but this approach fails to provide insight into solid tumor initiation and progression. Complex diseases with long latencies, including Alzheimer’s and Parkinson’s, can be partially modeled using induced pluripotent stem cells (iPSCs). However, short-term culture in a dish cannot capture the lengthy process of disease progression in a living organism.

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