Liver gene transfer for metabolite detoxification in inherited metabolic diseases

Authors: Alfonso M. D’Alessio , Iolanda Boffa, Lucia De Stefano , Leandro R. Soria and Nicola Brunetti-Pierri
Year: 2024
Sources: FEBS Letter
Inherited metabolic disorders (IMDs) are a growing group of genetic diseases caused by defects in enzymes that mediate cellular metabolism, often resulting in the accumulation of toxic substrates. The liver is a highly metabolically active organ that hosts several thousands of chemical reactions. As such, it is an organ frequently affected in IMDs. In this article, we review current approaches for liver-directed gene-based therapy aimed at metabolite detoxification in a variety of IMDs. Moreover, we discuss current unresolved challenges in gene-based therapies for IMDs.
Category: journals