2024-02-01 09:26:32
ANPLogo Amsterdam UMC on the facade of the hospital
NOS Nieuws•vandaag, 10:26
Ten patients with a hereditary swelling disease have almost completely resolved their complaints through gene therapy at the Amsterdam UMC, among others. They had angioedema, where swelling can occur at unexpected times due to an error in the DNA. These can occur throughout the body.
For example, there is a risk of throat swelling that can cause patients to choke. People therefore live in fear that the next attack could be fatal.
The patients participated in a pilot for the gene therapy CRISPR-Cas at the Amsterdam UMC, the University of Auckland and Cambridge University Hospitals. They were put on an IV for a few hours on a one-off basis.
‘Totally overwhelming’
“This is completely overwhelming,” says Danny Cohn, internist and researcher at Amsterdam UMC NOS Radio 1 News. He speaks of a breakthrough. Cohn has been treating patients with angioedema for about seven years. “Until a few years ago, we could not have imagined that we could cure these patients with treatment.”
Genetic scissors
The CRISPR-Cas gene therapy is a technique for modifying DNA. “CRISPR-Cas is also called the ‘genetic scissors,’” says Cohn. It is a way to precisely cut and paste DNA.
In patients with angioedema, a certain protein is produced in the liver that causes swelling in the body. With gene therapy, the gene that produces the protein is ‘cut’ in the liver cell via an infusion.
95 percent less swelling
The treatment reduced the concentration of the protein by 95 percent. The patients also had 95 percent less swelling. All patients have had no seizures since treatment.
The researchers have indications that the patients are rid of the disease for good, says Cohn. “Even if new liver cells are created, the protein does not return.” People can still pass on the genetic disease to their offspring. It is estimated that one in 50,000 people worldwide has angioedema.
Of the ten patients in the study, two were treated at the Amsterdam UMC. The others received the gene therapy at the University of Auckland and Cambridge University Hospitals. More patients have now been treated with CRISPR-Cas in the Amsterdam UMC.
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