Liver gene transfer for metabolite detoxification in inherited metabolic diseases.

D'Alessio, Alfonso M; Boffa, Iolanda; De Stefano, Lucia; Soria, Leandro R; Brunetti-Pierri, Nicola

D'Alessio, Alfonso M; Boffa, Iolanda; De Stefano, Lucia; Soria, Leandro R; Brunetti-Pierri, Nicola.

Afiliación

D'Alessio AM; Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.
Boffa I; Scuola Superiore Meridionale (SSM, School of Advanced Studies), Genomics and Experimental Medicine Program, University of Naples Federico II, Naples, Italy.
De Stefano L; Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.
Soria LR; Azienda Ospedaliera Universitaria Federico II, Naples, Italy.
Brunetti-Pierri N; Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.

FEBS Lett ; 2024 Jun 17.

Article en En | MEDLINE | ID: mdl-38884367

ABSTRACT

ABSTRACT

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.

Palabras clave

AAV; gene therapy; inherited metabolic disorders; lipid nanoparticles; liver; metabolite detoxification

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: FEBS Lett Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Reino Unido

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google