Article

Humanized mice bearing CRISPR/Cas9 Disruption of Signal Transducer and Activator of Transcription 1 (STAT1) to Model Primary Immunodeficiency

2022-06-21

Abstract excerpt

<h4>Background</h4> The search for a single, pathogenic genetic variant in a patient suspected to have a monogenic inborn error of immunity (IEI) often reveals a multitude of rare variants of unknown significance (VUS). Distinguishing which VUS is disease-causing versus the irrelevant, rare variants from the genetic background is slow and difficult. Advances in gene editing technology, particularly CRISPR/Cas9, p...

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Literature Corpus work: b205f909-4bc0-5e84-8ac1-4b3dc1a2a51d
DOI: 10.1101/2022.06.20.496920

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