Long-term Treatment Success Using Gene Therapy to Correct a Lethal Metabolic Disorder

PRLog - Oct. 9, 2014 - NEW ROCHELLE, N.Y. -- Excessive and often lethal blood levels of bilirubin can result from mutations in a single gene that are the cause of the metabolic disease known as Crigler-Najjar syndrome type 1 . A new gene therapy approach to correcting this metabolic error achieved significant, long-lasting reductions in bilirubin levels in a mouse model of CNS1 and is described in an Open Access article in Human Gene Therapy , a peer-reviewed journal from Mary Ann Liebert, Inc., publishers .



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