Article
Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects.
Clinical immunology (Orlando, Fla.) - 1 Jan 2024
Ameratunga Rohan, Leung Euphemia, Woon See-Tarn, Lea Edward, Allan Caroline, Chan Lydia, Longhurst Hilary, Steele Richard, Snell Russell, Lehnert Klaus
Abstract excerpt
The original CRISPR Cas9 gene editing system and subsequent innovations offers unprecedented opportunities to correct severe genetic defects including those causing Primary Immunodeficiencies (PIDs). Common Variable Immunodeficiency Disorders (CVID) are the most frequent symptomatic PID in adults and children. Unlike many other PIDs, patients meeting CVID criteria do not have a definable genetic defect and cannot...
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