CRISPR/Cas9-engineered avatar mouse model of rare disease paves the way for targeted therapy

Glass laboratory flasks on light table

An international research team led by Dr Ana GuadaƱo at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into the mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior of the cell.

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