Murphy & colleagues reported on a patient with WHIM syndrome who was cured of the disease by a spontaneous somatic genetic event that deleted the mutant CXCR4 allele in a single hematopoietic stem cell.

Here, the team now show CRISPR silencing of the Cxcr4 overactive disease allele corrects leukopenia in a murine model of WHIM syndrome, demonstrating a new therapeutic strategy for dominant immune disorders.

Molecular Signaling Section, Laboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, Maryland, USA.