Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder
Hereditary angioedema, or HAE, is a rare disease that affects about one in 50,000 people. It is caused by a genetic mutation that leaves patients with leaky blood vessels, causing erratic bouts of swelling. Dr. Hilary Longhurst, the principal investigator at New Zealand’s Te Toka Tumai, Auckland City hospital, said the new single-dose therapy appeared to provide a permanent cure for her patients’ “very disabling symptoms”.