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The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy

27 Mar 2025

Abstract excerpt

Gene therapy has revolutionized modern medicine by offering innovative treatments for genetic and acquired diseases. The liver has been and continues as a prime target for in vivo gene therapy due to its essential biological functions, vascular access to the major target cell (hepatocytes), and relatively immunotolerant environment. Adeno-associated virus (AAV) vectors have become the cornerstone of...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Identifiers

PMID: 40156191
DOI: 10.1016/j.ymthe.2025.03.041

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