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 port...

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Hauptverfasser: 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
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creator 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
description 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
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subjects HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
title Cells and non-human animals engineered to express ADAR1 and uses thereof
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