A single dose of gene therapy was enough to stop the painful swelling attacks caused by angiodema.
Gene therapy has transformed the lives of people with a genetic disorder that causes painful and unpredictable swelling attacks.
Patients treated with a single dose of gene therapy Crispr-Cas9 showed little sign of further symptoms."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.
"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. "I am very positive about the future. I am now volunteering where I can meet like-minded people. The freedom has opened up my world and improved my self-confidence."Crispr-Cas9 is a DNA cutting and pasting system that scientists have borrowed from nature. Bacteria use it to protect themselves against foreign DNA from viruses. Scientists have been using it in the lab to target and cut out faulty DNA in human cells that cause illnesses.
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