Angiodema: Gene therapy blocks painful hereditary disorder

Gene therapy has transformed the lives of people with a genetic disorder that causes painful and unpredictable swelling attacks.

Angiodema, thought to affect 50,000 people worldwide, can be seriously debilitating, affect airways, and occasionally prove fatal.

Patients treated with a single dose of gene therapy Crispr-Cas9 showed little sign of further symptoms.

The therapy also has big potential as a treatment for other genetic conditions.

“It looks as if the single-dose treatment will provide a permanent cure for my hereditary angioedema patients’ very disabling symptoms,” principal investigator Dr Hilary Longhurst, a clinical immunologist at Auckland hospital, said.

Angiodema causes swelling in the soft tissues and can affect many parts of the body.

Cleveland Firman, from Suffolk, who received the treatment in the UK said: “The randomness, unpredictability and potential severity of the attacks has made trying to live my life almost impossible. I spent my life constantly wondering if my next attack would be severe.

“The swellings are painful and disfiguring. I was embarrassed to go out in case of an attack. I’ve been hospitalised with swellings on my neck and throat that have affected my ability to breathe.

“Since the treatment, I’ve not had a single attack. I’ve had a radical improvement in my physical and mental wellbeing.

“I am very positive about the future. I am now volunteering where I can meet like-minded people. The freedom has opened up