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.

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