Gene Therapy for Metabolic Diseases of the Liver


Significant advances have been made in the field of liver-directed gene therapy. Many diseases are potential targets for gene therapy, including diseases that have exclusive liver involvement and those with systemic manifestations as a result of defective protein synthesis from the liver. Examples are Crigler-Najjar syndrome type 1, α1-antitrypsin… (More)
DOI: 10.2165/00063030-200013030-00003


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