Researchers Develop Mouse Model Incorporating Human Gene (MAPT) Involved in Alzheimer’s Disease

In research that helps scientists better understand and explore treatments for diseases like Alzheimer’s, scientists have developed a line of mice in which the mouse version of the Alzheimer’s-associated MAPT gene has been fully replaced by the human version of the gene. In this new animal model, known as a full-gene-replacement model, the MAPT gene will function the same way it does in humans, allowing researchers to more accurately develop and evaluate genetic therapies. The research was presented on October 16 at the American Society of Human Genetics (ASHG) 2019 Annual Meeting in Houston, Texas (October 15-19). The presentation abstract is titled “Moving Human Genetics into the Mouse: Full Human Gene-Replacement Models.” Scientists have long studied human genes in mice and other animal models, usually by finding and manipulating the animal’s version of the human gene being studied, explained Michael Koob, PhD, Associate Professor at the University of Minnesota, who presented the work. “However, mice have different genes than people, and even if the gene’s function is the same, its sequence is different,” Dr. Koob said. For this reason, animal model work typically involves a great deal of trial and error, and it requires researchers to make assumptions about why and how a genetic change leads to the observed changes. In addition, drawing conclusions about the role of the human version of the gene in humans – and building on this knowledge by developing therapies – is difficult and prone to error, and the findings do not always translate.
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