The New England Journal of Medicine has published new correspondence on how well gene therapy holds up over time in patients with Crigler–Najjar syndrome, a rare inherited liver disorder.
The *New England Journal of Medicine* posted findings on the durability of gene therapy in patients living with Crigler–Najjar syndrome (a genetic condition that stops the liver producing a protein needed to break down bilirubin, a yellow substance made when red blood cells are recycled). Without that protein, bilirubin builds up in the blood and can cause serious harm, including brain damage.
Gene therapy works by delivering a corrected copy of the faulty gene into the body’s cells. The question researchers have been tracking is how long that correction holds — and whether patients continue to benefit months or years after treatment.
The correspondence, published under the journal’s gastroenterology and genetics categories, adds to a growing body of evidence on gene therapy’s long-term performance in rare inherited conditions. Crigler–Najjar syndrome is serious enough that some patients currently spend up to 12 hours a day under special blue lights (phototherapy) to keep bilirubin at safe levels — a demanding routine that a successful gene therapy could reduce or replace.
The correspondence reports four-year follow-up data in five patients, according to the NEJM record.
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*This article is based on a published journal correspondence. It does not constitute medical advice. Anyone with concerns about liver conditions or inherited disorders should speak to their GP or call NHS 111.*
Source: @NEJM