Given the heterogeneity and rarity of inborn errors of immunity (IEIs), management guidelines that deal with primary immunodeficiency as a whole are rare. The most recent general guideline, the ...

Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first Phase III study of an in vivo CRISPR therapy. In this large-scale, double-blind trial, 80 ...

A single treatment with a CRISPR-Cas9 based gene editing therapy is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful ...

We are providing an unedited version of this manuscript to give early access to its findings. Before final publication, the manuscript will undergo further editing. Please note there may be errors ...