Gene therapy blocks the painful hereditary disorder Angiodema

Gene therapy blocks the painful hereditary disorder Angiodema
Acute sleeping sickness parasite

Angiodema, which plagues the lives of more than 50,000 people, congests people’s airways, and occasionally is fatal.

But now, gene therapy is blocking this painful hereditary disorder. The BBC reports that “patients treated with a single dose of gene therapy Crispr-Cas9 showed little sign of further symptoms.”

Similar therapies could be used as a treatment for other devastating genetic conditions.

“It looks as if the single-dose treatment will provide a permanent cure for my hereditary angioedema patients’ very disabling symptoms,” says Dr Hilary Longhurst, a clinical immunologist in New Zealand.

Angiodema causes severe swelling in soft tissues and can afflict many areas of the body.

Cleveland Firman, who received the treatment in England, lauded it, saying, “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 my world.”

As the BBC notes,

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.

However, the edits are not always perfect and the cuts can end up in the wrong place.

In the trial, published in the New England Journal of Medicine, angiodema symptoms were reduced by 95% among a small group of patients from the UK, New Zealand, and the Netherlands given a single infusion – targeted at cells in the liver – to reduce the KLKB1 gene’s ability to produce plasma prekallikrein.

Just one of the patients continued to show symptoms, which were only minor.

Dr Padmalal Gurugama of Cambridge University Hospital says the therapy will enormously improve people’s lives. “Hereditary angioedema can cause patients severe swellings and intense pain, which can be life-threatening as well as restricting normal activities, such as going to work or school. Because it is often misdiagnosed, many patients undergo unnecessary treatments and invasive procedures.”

Recently, a treatment was discovered for sleeping sickness, a horrific disease that kills 50,000 to 500,000 people per year.

Scientists recently discovered a new antibiotic that can kill drug-resistant bacteria.

Coffee grounds may hold the secret to curing Alzheimer’s, Parkinson’s, and Huntington’s diseases.

A virus is being used to cure deafness in new gene therapy. Researchers also discovered that a plant virus could be used to save crops from root-eating pests.

Doctors overseas are using artificial intelligence to detect cases of breast cancer more effectively. A new ultrasound therapy could help treat cancer and Alzheimer’s disease.

Doctors recently used a surgical robot to carry out incredibly complicated spinal surgery. Doctors also recently did the first robotic liver transplant in America.

LU Staff

LU Staff

Promoting and defending liberty, as defined by the nation’s founders, requires both facts and philosophical thought, transcending all elements of our culture, from partisan politics to social issues, the workings of government, and entertainment and off-duty interests. Liberty Unyielding is committed to bringing together voices that will fuel the flame of liberty, with a dialogue that is lively and informative.

Comments

For your convenience, you may leave commments below using Disqus. If Disqus is not appearing for you, please disable AdBlock to leave a comment.