Gene-editing offers hope for people with hereditary disorder

Dr Padmalal Gurugama


A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy.

The patients have hereditary angioedema, a genetic disorder characterised by severe, painful and unpredictable swelling attacks. These interfere with daily life and can affect airways and prove fatal.

Now researchers from Cambridge University Hospitals, University of Auckland and Amsterdam University Medical Center have successfully treated more than ten patients with the CRISPR/Cas9 therapy, with interim results just published in a leading journal.

The NIHR Cambridge CRF worked with the UK Principal Investigator, Dr Padmalal Gurugama, consultant in clinical immunology and allergy at Cambridge University Hospitals, and his team to deliver this ground-breaking treatment to a local patient, who says it has transformed his life.

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