Article

T cell correction pipeline for Inborn Errors of Immunity

2024-09-06

Abstract excerpt

CRISPR/Cas9 gene editing technology is a promising tool for correcting pathogenic variants for autologous cell therapies for Inborn Errors of Immunity (IEI). The present IEI correction strategies mainly focus on the knock-in of therapeutic cDNAs, or knockout of the disease-causing gene when feasible. These strategies address many single-gene defects but may disrupt gene expression and require significant optimizat...

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Literature Corpus work: a7c467be-9484-5ae4-978c-7f68dd48bc22
DOI: 10.1101/2024.09.03.610811

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