Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.
Gene therapy for inherited diseases of liver metabolism / Piccolo, P; BRUNETTI PIERRI, Nicola. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - 26:(2015), pp. 186-192. [10.1089/hum.2015.029]
Gene therapy for inherited diseases of liver metabolism.
Piccolo P;BRUNETTI PIERRI, NICOLA
2015
Abstract
Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.File in questo prodotto:
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