The future of gene therapy for inborn errors of metabolism: insights into liver-targeted innovations

Expert Opinion on Biological Therapy 2026 Open access

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Authors: Gloria Gonzalez-Aseguinolaza, Pasquale Piccolo
DOI: 10.1080/14712598.2026.2618488
Cited by: 1
References: 14

Abstract

1. Liver-directed gene therapy has progressed from conceptual promise to clinical reality for several inborn errors of metabolism (IEMs). The liver is a particularly attractive target because it ho...

Fields of Study & Subjects

Metabolism and Genetic Disorders Virus-based gene therapy research Genomics and Rare Diseases

Publication details

Journal / source
Expert Opinion on Biological Therapy
Publisher
Informa UK Limited
Volume
26
Issue
1
Pages
11–14
Publication date
2 January 2026
Language
EN