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

• Main Authors: YANG HAILIN, VARGHESE CHANDRA, PRASHANT. MONIAN, YU HUI, KANDASAMUTHU. KANDASAMUTHU, LU GENLIANG, CHIKDU SHAKTI SHIVALILA, GODFREY JACK DAVID, BLAY DAVID JOHN, SHIMIZU MAMORU, STANDLEY STEFANIE MICHELLE, APOLONI LUCIANO ENRIQUE, IWAMOTO NAOKI, BUTLER DAVID CHARLES DONNELL, MALAPPAN SUBRAMANIAN
• Format: Patent
• Language: Chinese; English
• Published: 18.04.2023
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

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