Cells and non-human animals engineered to express ADAR1 and uses thereof

The disclosure provides, among other things, cells and non-human animals engineered to express an ADAR1 polypeptide, or a characteristic portion thereof. In some embodiments, the disclosure provides cells and non-human animals engineered to express a human ADAR1 polypeptide, or a characteristic portion thereof. In some embodiments, the non-human animals are genetically modified rodents, such as mice, rats, and the like. In some embodiments, the non-human animal is a mouse. In some embodiments, the disclosure provides techniques for assessing an agent comprising administering the agent to a cell or non-human animal engineered to express an ADAR1 polypeptide, or a characteristic portion thereof. In some embodiments, such cells or non-human animals are engineered to express a human ADAR1 polypeptide, or a characteristic portion thereof. In some embodiments, the medicament is a pharmaceutical formulation. In some embodiments, the agent is or comprises an oligonucleotide. 除其他外，本披露提供了经工程化为表达ADAR1多肽或其特征性部分的细胞和非人动物。在