Gene editing offers hope to patients with inherited disorders: Study

Los Angeles [US], February 5 (ANI): According to the lead researcher, a single treatment with a groundbreaking gene-editing therapy changed the lives of a group of patients suffering from a hereditary ailment.

Patients from New Zealand, the Netherlands, and the United Kingdom have hereditary angioedema, a genetic disease that causes severe, painful, and sudden swelling attacks. These disturb normal activities and have the potential to cause airway damage and death.

Researchers from the University of Auckland, Amsterdam University Medical Center, and Cambridge University Hospitals have successfully treated over ten patients with CRISPR/Cas9 technology, with preliminary findings just published in a leading journal.

“It looks as if the single-dose treatment will provide a permanent cure for my hereditary angioedema patients’ very disabling symptoms,” said principal investigator Dr Hilary Longhurst, who is both a clinical immunologist at Auckland Hospital Te Toku Tumai and an honorary associate professor at the University of Auckland.

“Plus, of course, there is huge potential for development of similar CRISPR/Cas9 treatments for other genetic disorders.”

Globally, it is estimated one in 50,000 people have hereditary angioedema, however, because it is rare, it is often not correctly diagnosed.

In the phase one study, there were no serious or lasting side effects from the single infusion, which took place over two to four hours under clinical supervision from late 2021 and onwards.