CELLS AND NON-HUMAN ANIMALS ENGINEERED TO EXPRESS ADAR1 AND USES THEREOF

CELLS AND NON-HUMAN ANIMALS ENGINEERED TO EXPRESS ADAR1 AND USES THEREOF

Among other things, the present disclosure provides cells and non-human animals engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, the present disclosure provides cells and non-human animals engineered to express a human ADAR1 polypeptide or a charac...

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Bibliographische Detailangaben
Hauptverfasser: MARAPPAN, Subramanian, BUTLER, David, Charles, Donnell, APPONI, Luciano, Henrique, BOULAY, David, John, SHIVALILA, Chikdu Shakti, MONIAN, Prashant, IWAMOTO, Naoki, YU, Hui, STANDLEY, Stephany, Michelle, SHIMIZU, Mamoru, VARGEESE, Chandra, LU, Genliang, KANDASAMY, Pachamuthu, GODFREY, Jack, David, YANG, Hailin
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Beschreibung
Zusammenfassung:Among other things, the present disclosure provides cells and non-human animals engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, the present disclosure provides cells and non-human animals engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, non-human animals are genetically modified rodents such as mice, rat, etc. In some embodiments, non-human animals are mice. In some embodiments, the present disclosure provides technologies 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 a cell or non-human animal is engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, an agent is a pharmaceutical agent. In some embodiments, an agent is or comprises an oligonucleotide.