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 ...

Hereditary angioedema (HAE) is a rare, autosomal dominant disorder defined by episodic, potentially life‐threatening swellings that can affect the skin, gastrointestinal tract, and upper airways.

This Review reports on a series of recent discoveries that have added to the characterization of the molecular and cellular basis of primary immunodeficiencies (PIDs) in humans. A total of more than ...

Researchers from Children's Hospital of Philadelphia (CHOP) and the Clinic for Special Children found that complement factor I (CFI) deficiency, an ultra-rare genetic disorder that can cause ...

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 ...

Iron deficiency in pregnant mice may lead to the development of ovaries in a small proportion of offspring carrying XY chromosomes, which typically determine male sex. The findings, published in ...

Scientists at Gladstone Institutes have flipped the traditional approach to finding potential treatments for deadly diseases.