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Liver‐directed gene therapy for inherited metabolic diseases

1 Jan 2024

Abstract excerpt

Gene therapy clinical trials are rapidly expanding for inherited metabolic liver diseases whilst two gene therapy products have now been approved for liver based monogenic disorders. Liver-directed gene therapy has recently become an option for treatment of haemophilias and is likely to become one of the favoured therapeutic strategies for inherited metabolic liver diseases in the near future. In this review, we...

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PMID: 38171926
DOI: 10.1002/jimd.12709

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