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 ...

TWO inborn errors of the complement system of man have been well documented: hereditary deficiency of the serum inhibitor of the activated first component of complement (C′1a) and hereditary ...

Hereditary angioedema (HAE) is a rare genetic disorder that causes episodes of severe swelling throughout your body. It is caused by abnormal immune responses. But unlike allergic swelling, HAE is not ...